0001628280-22-028168 | 10-Q | XBRL Viewer

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Cover - shares	9 Months Ended
Cover - shares	Sep. 30, 2022	Oct. 31, 2022
Cover [Abstract]
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Sep. 30, 2022
Document Transition Report	false
Entity File Number	000-30171
Entity Registrant Name	SANGAMO THERAPEUTICS, INC.
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	68-0359556
Entity Address, Address Line One	7000 Marina Blvd.
Entity Address, City or Town	Brisbane
Entity Address, State or Province	CA
Entity Address, Postal Zip Code	94005
City Area Code	510
Local Phone Number	970-6000
Title of 12(b) Security	Common Stock, par value $0.01 per share
Trading Symbol	SGMO
Security Exchange Name	NASDAQ
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Entity Shell Company	false
Entity Common Stock, Shares Outstanding		163,880,007
Amendment Flag	false
Document Fiscal Year Focus	2022
Document Fiscal Period Focus	Q3
Entity Central Index Key	0001001233
Current Fiscal Year End Date	--12-31

CONDENSED CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands	Sep. 30, 2022	Dec. 31, 2021
Current assets:
Cash and cash equivalents	$ 94,570	$ 178,872
Marketable securities	218,998	197,676
Interest receivable	524	349
Accounts receivable	4,522	6,013
Prepaid expenses and other current assets	18,007	15,859
Total current assets	336,621	398,769
Marketable securities, non-current	36,700	88,169
Property and equipment, net	55,986	51,523
Intangible assets	46,439	53,760
Goodwill	34,511	39,702
Operating lease right-of-use assets	64,956	73,181
Other non-current assets	17,200	15,319
Restricted cash	1,500	1,500
Total assets	593,913	721,923
Current liabilities:
Accounts payable	17,380	9,759
Accrued compensation and employee benefits	19,045	20,840
Other accrued liabilities	14,046	11,577
Deferred revenues	71,890	85,711
Total current liabilities	122,361	127,887
Deferred revenues, non-current	115,641	166,776
Long-term portion of lease liabilities	39,705	44,055
Deferred income tax	5,740	6,645
Other non-current liabilities	1,312	1,217
Total liabilities	284,759	346,580
Commitments and contingencies
Stockholders’ equity:
Preferred stock	0	0
Common stock	1,619	1,459
Additional paid-in capital	1,423,105	1,334,138
Accumulated deficit	(1,096,572)	(956,267)
Accumulated other comprehensive loss	(18,998)	(3,987)
Total stockholders’ equity	309,154	375,343
Total liabilities and stockholders’ equity	$ 593,913	$ 721,923

CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS - USD ($) shares in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2022	Sep. 30, 2021	Sep. 30, 2022	Sep. 30, 2021
Income Statement [Abstract]
Revenues	$ 26,460,000	$ 28,563,000	$ 84,069,000	$ 82,715,000
Operating expenses:
Research and development	65,116,000	62,498,000	183,719,000	179,018,000
General and administrative	16,238,000	14,501,000	46,239,000	47,135,000
Total operating expenses	81,354,000	76,999,000	229,958,000	226,153,000
Loss from operations	(54,894,000)	(48,436,000)	(145,889,000)	(143,438,000)
Interest and other income, net	1,769,000	834,000	5,754,000	3,010,000
Loss before income taxes	(53,125,000)	(47,602,000)	(140,135,000)	(140,428,000)
Income tax expense	30,000	86,000	170,000	373,000
Net loss	(53,155,000)	(47,688,000)	(140,305,000)	(140,801,000)
Net loss attributable to non-controlling interest	0	0	0	(11,000)
Net loss attributable to Sangamo Therapeutics, Inc. stockholders, basic	(53,155,000)	(47,688,000)	(140,305,000)	(140,790,000)
Net loss attributable to Sangamo Therapeutics, Inc. stockholders, diluted	$ (53,155,000)	$ (47,688,000)	$ (140,305,000)	$ (140,790,000)
Earnings per share, basic (in dollars per share)	$ (0.34)	$ (0.33)	$ (0.93)	$ (0.98)
Earnings per share, diluted (in dollars per share)	$ (0.34)	$ (0.33)	$ (0.93)	$ (0.98)
Shares used in computing basic net loss per share attributable to Sangamo Therapeutics, Inc. stockholders (in shares)	158,042	145,399	150,850	144,173
Shares used in computing diluted net loss per share attributable to Sangamo Therapeutics, Inc. stockholders (in shares)	158,042	145,399	150,850	144,173

CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2022	Sep. 30, 2021	Sep. 30, 2022	Sep. 30, 2021
Statement of Comprehensive Income [Abstract]
Net loss	$ (53,155)	$ (47,688)	$ (140,305)	$ (140,801)
Foreign currency translation adjustment	(6,028)	(2,565)	(14,040)	(6,006)
Net pension gains	55	0	130	0
Unrealized gain (loss) on marketable securities, net of tax	135	(45)	(1,101)	(36)
Comprehensive loss	(58,993)	(50,298)	(155,316)	(146,843)
Comprehensive loss attributable to non-controlling interest	0	0	0	(11)
Comprehensive loss attributable to Sangamo Therapeutics, Inc.	$ (58,993)	$ (50,298)	$ (155,316)	$ (146,832)

- Definition

Amount after tax of increase (decrease) in equity from transactions and other events and circumstances from net income (loss) and other comprehensive income (loss), attributable to noncontrolling interests. Excludes changes in equity resulting from investments by owners and distributions to owners.

+ References + Details

- Definition

Amount after tax of increase (decrease) in equity from transactions and other events and circumstances from net income and other comprehensive income. Excludes changes in equity resulting from investments by owners and distributions to owners.

+ References + Details

- Definition

The consolidated profit or loss for the period, net of income taxes, including the portion attributable to the noncontrolling interest.

+ References

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+ Details

CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY - USD ($) $ in Thousands	Total	Common Stock	Additional Paid-in Capital	Accumulated Deficit	Accumulated Other Comprehensive (Loss) Income	Non- Controlling Interest
Beginning balance (in shares) at Dec. 31, 2020		142,063,203
Beginning balance at Dec. 31, 2020	$ 497,366	$ 1,421	$ 1,269,375	$ (777,981)	$ 5,419	$ (868)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuance of common stock in connection with at-the-market offering, net of offering expenses (in shares)		2,007,932
Issuance of common stock in connection with at-the-market offering, net of offering expenses	27,099	$ 20	27,079
Issuance of common stock upon exercise of stock options and in connection with restricted stock units, net of tax (in shares)		1,200,871
Issuance of common stock upon exercise of stock options and in connection with restricted stock units, net of tax	1,594	$ 12	1,582
Issuance of common stock under employee stock purchase plan (in shares)		244,570
Issuance of common stock under employee stock purchase plan	2,054	$ 2	2,052
Stock-based compensation	24,880		24,880
Acquisition of additional shares of Sangamo France	(134)		(70)			(64)
Foreign currency translation adjustment	(6,006)				(6,006)
Net pension gains	0
Unrealized gain (loss) on marketable securities, net of tax	(36)				(36)
Buy-out of non-controlling interest	(943)		(943)			943
Net loss	(140,801)			(140,790)		(11)
Ending balance (in shares) at Sep. 30, 2021		145,516,576
Ending balance at Sep. 30, 2021	406,016	$ 1,455	1,323,955	(918,771)	(623)	0
Beginning balance (in shares) at Jun. 30, 2021		145,106,901
Beginning balance at Jun. 30, 2021	445,457	$ 1,451	1,313,102	(871,083)	1,987	0
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuance of common stock in connection with at-the-market offering, net of offering expenses (in shares)		202,705
Issuance of common stock in connection with at-the-market offering, net of offering expenses	2,367	$ 2	2,365
Issuance of common stock upon exercise of stock options and in connection with restricted stock units, net of tax (in shares)		206,970
Issuance of common stock upon exercise of stock options and in connection with restricted stock units, net of tax	617	$ 2	615
Stock-based compensation	7,873		7,873
Foreign currency translation adjustment	(2,565)				(2,565)
Net pension gains	0
Unrealized gain (loss) on marketable securities, net of tax	(45)				(45)
Net loss	(47,688)			(47,688)		0
Ending balance (in shares) at Sep. 30, 2021		145,516,576
Ending balance at Sep. 30, 2021	406,016	$ 1,455	1,323,955	(918,771)	(623)	$ 0
Beginning balance (in shares) at Dec. 31, 2021		145,921,530
Beginning balance at Dec. 31, 2021	375,343	$ 1,459	1,334,138	(956,267)	(3,987)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuance of common stock in connection with at-the-market offering, net of offering expenses (in shares)		14,711,770
Issuance of common stock in connection with at-the-market offering, net of offering expenses	66,448	$ 147	66,301
Issuance of common stock upon exercise of stock options and in connection with restricted stock units, net of tax (in shares)		925,455
Issuance of common stock upon exercise of stock options and in connection with restricted stock units, net of tax	(1,838)	$ 9	(1,847)
Issuance of common stock under employee stock purchase plan (in shares)		359,468
Issuance of common stock under employee stock purchase plan	1,115	$ 4	1,111
Stock-based compensation	23,402		23,402
Foreign currency translation adjustment	(14,040)				(14,040)
Net pension gains	130				130
Unrealized gain (loss) on marketable securities, net of tax	(1,101)				(1,101)
Buy-out of non-controlling interest	0
Net loss	(140,305)			(140,305)
Ending balance (in shares) at Sep. 30, 2022		161,918,223
Ending balance at Sep. 30, 2022	309,154	$ 1,619	1,423,105	(1,096,572)	(18,998)
Beginning balance (in shares) at Jun. 30, 2022		153,352,502
Beginning balance at Jun. 30, 2022	318,281	$ 1,534	1,373,324	(1,043,417)	(13,160)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Issuance of common stock in connection with at-the-market offering, net of offering expenses (in shares)		8,483,104
Issuance of common stock in connection with at-the-market offering, net of offering expenses	42,174	$ 85	42,089
Issuance of common stock upon exercise of stock options and in connection with restricted stock units, net of tax (in shares)		82,617
Issuance of common stock upon exercise of stock options and in connection with restricted stock units, net of tax	(101)		(101)
Stock-based compensation	7,793		7,793
Foreign currency translation adjustment	(6,028)				(6,028)
Net pension gains	55				55
Unrealized gain (loss) on marketable securities, net of tax	135				135
Net loss	(53,155)			(53,155)
Ending balance (in shares) at Sep. 30, 2022		161,918,223
Ending balance at Sep. 30, 2022	$ 309,154	$ 1,619	$ 1,423,105	$ (1,096,572)	$ (18,998)

CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands	9 Months Ended
	Sep. 30, 2022	Sep. 30, 2021
Operating Activities:
Net loss	$ (140,305)	$ (140,801)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization	8,765	6,675
Amortization of (discount) premium on marketable securities	(324)	2,394
Amortization and other changes in operating lease right-of-use assets	6,357	6,104
Gain on free shares	0	(18)
Stock-based compensation	23,402	24,880
Gain on disposal of property and equipment	0	(30)
Net changes in operating assets and liabilities:
Interest receivable	(175)	415
Accounts receivable	1,491	(2,743)
Prepaid expenses and other assets	(5,848)	(6,467)
Accounts payable and other accrued liabilities	9,443	(4,699)
Accrued compensation and employee benefits	(1,225)	(1,932)
Deferred revenues	(64,956)	(62,292)
Lease liabilities	(3,298)	(3,227)
Other non-current liabilities	95	1,204
Net cash used in operating activities	(166,578)	(180,537)
Investing Activities:
Purchases of marketable securities	(225,621)	(300,387)
Maturities of marketable securities	254,990	509,620
Sales of marketable securities	0	6,870
Purchases of property and equipment	(12,697)	(20,420)
Purchase of additional Sangamo France shares	0	(119)
Net cash provided by investing activities	16,672	195,564
Financing Activities:
Taxes paid related to net share settlement of equity awards	(1,962)	(2,988)
Proceeds from exercise of stock options	124	4,582
Net cash provided by financing activities	65,125	30,746
Effect of exchange rate changes on cash, cash equivalents, and restricted cash	479	(148)
Net (decrease) increase in cash, cash equivalents, and restricted cash	(84,302)	45,625
Cash, cash equivalents, and restricted cash, beginning of period	180,372	132,829
Cash, cash equivalents, and restricted cash, end of period	96,070	178,454
Supplemental cash flow disclosures:
Property and equipment included in unpaid liabilities	3,927	650
Right-of-use assets obtained in exchange for lease obligations	0	1,349
Tenant improvement allowance included in contra-lease liability	1,531	0
Buy-out of non-controlling interest	0	943
At the Market Offering
Financing Activities:
Proceeds from at-the market offering/ issuance of common stock	65,848	27,099
Employee Stock
Financing Activities:
Proceeds from at-the market offering/ issuance of common stock	$ 1,115	$ 2,053

ORGANIZATION, BASIS OF PRESENTATION, AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES

9 Months Ended

Sep. 30, 2022

Accounting Policies [Abstract]

ORGANIZATION, BASIS OF PRESENTATION, AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES

ORGANIZATION, BASIS OF PRESENTATION AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES

Organization and Description of Business

Sangamo Therapeutics, Inc. (“Sangamo” or “the Company”) was incorporated in the State of Delaware in June 1995 and changed its name from Sangamo Biosciences, Inc. in January 2017. Sangamo is a clinical-stage genomic medicine company committed to translating ground-breaking science into medicines that transform the lives of patients with serious diseases.

Basis of Presentation

The accompanying unaudited Condensed Consolidated Financial Statements have been prepared in accordance with generally accepted accounting principles in the United States of America (“U.S. GAAP”) for interim financial information and pursuant to the rules and regulations of the Securities and Exchange Commission (“SEC”). Accordingly, they do not include all of the information and footnotes required by U.S. GAAP for complete financial statements. In the opinion of management, all adjustments (consisting only of normal recurring adjustments) considered necessary for a fair presentation of these financial statements for the periods presented have been included. Operating results for the three and nine months ended September 30, 2022 are not necessarily indicative of the results that may be expected for the year ending December 31, 2022. The Condensed Consolidated Balance Sheet data at December 31, 2021 was derived from the audited Consolidated Financial Statements included in Sangamo’s Annual Report on Form 10-K for the year ended December 31, 2021 (the “2021 Annual Report”) as filed with the SEC on February 24, 2022.

The accompanying Condensed Consolidated Financial Statements include the accounts of the Company and its subsidiaries. All intercompany balances and transactions have been eliminated in the Condensed Consolidated Financial Statements. For consolidated entities where the Company owns or is exposed to less than 100% of the economics, the Company records net loss attributable to non-controlling interests on its Condensed Consolidated Statements of Operations equal to the percentage of the economic or ownership interest retained in such entities by the respective non-controlling parties.

The accompanying Condensed Consolidated Financial Statements and related financial information should be read together with the audited Consolidated Financial Statements and footnotes for the year ended December 31, 2021, included in the 2021 Annual Report.

Liquidity and Management’s Plan

Sangamo is currently working on a number of long-term development projects that involve experimental technologies. The projects may require several years and substantial expenditures to complete and ultimately may be unsuccessful. The Company plans to finance operations with available cash resources, collaborations and strategic partnerships funds, research grants and from the issuance of equity or debt securities. Sangamo believes that its available cash, cash equivalents, and marketable securities as of September 30, 2022, and expected future milestones and research services revenue from collaborations, strategic partnerships and research grants, will be adequate to fund its currently planned operations through at least the next 12 months from the date these Condensed Consolidated Financial Statements are issued. Sangamo will require substantial additional financial resources to complete the development and commercialization of its product candidates. Additional capital may not be available on terms acceptable to the Company, if at all. If adequate funds are not available, or if the terms of potential funding sources are unfavorable, the Company’s business and ability to develop its technology and therapeutic products would be harmed. Furthermore, any sales of additional equity securities may result in dilution to the Company’s stockholders, and any debt financing may include covenants that restrict the Company’s business.

Summary of Significant Accounting Policies

Use of Estimates

The preparation of Condensed Consolidated Financial Statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the amounts reported in the Condensed Consolidated Financial Statements and the accompanying notes. On an ongoing basis, management evaluates its estimates including critical accounting policies or estimates related to revenue recognition, clinical trial accruals, income taxes, fair value of assets and liabilities, including from acquisitions, and stock-based compensation. Estimates are based on historical experience and on various other market specific and other relevant assumptions that the Company believes to be reasonable under the circumstances, the results of which form the basis for

making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Actual results could differ from those estimates.

In September 2021, the Company recorded an adjustment to revenue related to a change in estimate in connection with the collaboration agreement with Sanofi S.A. (“Sanofi”) as a result of an increase in the project costs for its beta thalassemia program and the increase in project scope and the corresponding costs for its sickle cell disease program, both of which resulted in a decrease in the measure of proportional cumulative performance.

This adjustment decreased revenue by $2.5 million, increased net loss by $2.5 million and increased the Company's basic and diluted net loss per share by $0.02 for the three and nine months ended September 30, 2021.

Revenue Recognition

The Company accounts for its revenues pursuant to the provisions of Accounting Standards Codification (“ASC”) Topic 606, Revenue from Contracts with Customers (“ASC Topic 606”). The Company’s contract revenues are derived from collaboration agreements including licensing arrangements and research activity grants. Research and licensing agreements typically include upfront signing or license fees, cost reimbursements for research services, minimum sublicense fees, milestone payments and royalties on future licensees’ product sales. The Company has agreements with both fixed and variable consideration. Non-refundable upfront fees and funding of research and development activities are considered fixed, while milestone payments are generally identified as variable consideration. Sangamo’s research grants are typically multi-year agreements and provide for the reimbursement of qualified expenses for research and development as defined under the terms of the grant agreement. Revenues under research grant agreements are generally recognized when the related qualified research expenses are incurred. Deferred revenue primarily represents the portion of research or license payments received but not earned.

In determining the appropriate amount of revenue to be recognized as the Company fulfills its obligations under its agreements, the Company performs the following steps: (i) identification of the promised goods or services in the contract; (ii) determination of whether the promised goods or services are performance obligations, including whether they are distinct in the context of the contract; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations based on estimated selling prices; and (v) recognition of revenue when (or as) the Company satisfies each performance obligation.

A performance obligation is a promise in a contract to transfer a distinct good or service to the customer and is the unit of account in ASC Topic 606. The Company’s performance obligations include license rights, development services and services associated with regulatory submission and approval processes. Revenues from research services earned under collaboration agreements are generally recognized as revenue as the related services are provided. Revenues from non-refundable upfront fees are recognized over time either by measuring progress towards satisfaction of the relevant performance obligation, using the input method (i.e., cumulative actual costs incurred relative to total estimated costs) or on a straight-line basis when a performance obligation is expected to be satisfied evenly over a period of time (or when the entity has a stand-ready obligation). Significant management judgment is required to determine the level of effort required under an arrangement, and the period over which the Company expects to complete its performance obligations under the arrangement, which may include total internal personnel costs and external costs to be incurred as well as, in certain cases, the estimated stand-ready obligation period. Changes in these estimates can have a material effect on revenue recognized. If the Company cannot reasonably estimate when its performance obligations either are completed or become inconsequential, then revenue recognition is deferred until the Company can reasonably make such estimates. The Company includes the unconstrained amount of estimated variable consideration in the transaction price. The amount included in the transaction price is constrained to the amount for which it is probable that a significant reversal of cumulative revenue recognized will not occur. At the end of each subsequent reporting period, the Company re-evaluates the estimated variable consideration included in the transaction price and any related constraint and, if necessary, adjusts its estimate of the overall transaction price. Revenue is then recognized over the remaining estimated period of performance using the cumulative catch-up method. The estimated period of performance and project costs, such as personnel and manufacturing cost, are reviewed quarterly and adjusted, as needed, to reflect the Company’s current assumptions regarding the timing of its deliverables.

As part of the accounting for these arrangements, the Company must develop assumptions that require judgment to determine the stand-alone selling price of each performance obligation identified in the contract. The Company uses key assumptions to determine the stand-alone selling price, which may include forecasted revenues, development timelines, reimbursement rates for personnel costs, discount rates and probabilities of technical and regulatory success. Related costs and expenses under these arrangements have historically approximated the revenues recognized.

Revenues from major collaboration agreements and research activity grants as a percentage of total revenues were as follows:


	Three Months Ended September 30,				Nine Months Ended September 30,
	2022		2021		2022		2021
Novartis Institutes for BioMedical Research, Inc.	36	%	40	%	35	%	35	%
Biogen MA, Inc.	36	%	38	%	36	%	39	%
Kite Pharma, Inc.	26	%	22	%	23	%	23	%
Sanofi S.A.	—	%	(3)	%	4	%	2	%

Funds received from the Company’s collaboration partners are generally not refundable and are recorded as revenue as the Company fulfills its performance obligations, which are satisfied over time (i.e., stand ready obligations) or by using the input method (i.e., cumulative actual costs incurred relative to total estimated costs). Revenue is also recognized when the Company has incurred qualified research and development costs that are reimbursable from its collaboration partners and when there is reasonable assurance that such costs will be reimbursed. Any payments received from a collaboration partner in advance of the completion of the relevant performance obligation are recorded as deferred revenue.

Cash, Cash Equivalents, and Restricted Cash

Sangamo considers all highly liquid investments purchased with original maturities of three months or less at the purchase date to be cash equivalents. Cash and cash equivalents consist of cash, deposits in demand money market accounts and U.S. government-sponsored entity debt securities. Restricted cash consists of a letter of credit for $1.5 million, representing a deposit for the lease of the corporate headquarters in Brisbane, California.

A reconciliation of cash, cash equivalents, and restricted cash reported within the Condensed Consolidated Balance Sheets to the amounts reported within the accompanying Condensed Consolidated Statements of Cash Flows is as follows (in thousands):


	September 30, 2022		December 31, 2021		September 30, 2021		December 31, 2020
Cash and cash equivalents	$	94,570	$	178,872	$	176,954	$	131,329
Non-current restricted cash	1,500		1,500		1,500		1,500
Cash, cash equivalents, and restricted cash as reported within the accompanying Condensed Consolidated Statements of Cash Flows	$	96,070	$	180,372	$	178,454	$	132,829

Recently Adopted Accounting Pronouncements

None.

FAIR VALUE MEASUREMENTS

9 Months Ended

Sep. 30, 2022

Fair Value Disclosures [Abstract]

FAIR VALUE MEASUREMENTS

The Company measures certain financial assets and liabilities at fair value on a recurring basis, including cash equivalents, marketable securities, and the free shares asset. Fair value is determined based on a three-tier hierarchy under the authoritative guidance for fair value measurements and disclosures that prioritizes the inputs used in measuring fair value as follows:

Level 1: Unadjusted quoted prices in active markets that are accessible at the measurement date for identical, unrestricted assets or liabilities;

Level 2: Quoted prices in markets that are not active or inputs which are observable, either directly or indirectly, for substantially the full term of the asset or liability; and

Level 3: Prices or valuation techniques that require inputs that are both significant to the fair value measurements and unobservable (i.e., supported by little or no market activity).

The fair value measurements of the Company’s cash equivalents and marketable securities are identified at the following levels within the fair value hierarchy (in thousands):


	September 30, 2022
	Fair Value Measurements
	Total		Level 1		Level 2		Level 3
Assets:
Cash equivalents:
Money market funds	$	44,911	$	44,911	$	—	$	—


Total	44,911		44,911		—		—
Marketable securities:
U.S. government-sponsored entity debt securities	23,392		—		23,392		—
Commercial paper securities	120,386		—		120,386		—
Corporate debt securities	14,888		—		14,888		—
Asset-backed securities	37,773		—		37,773		—
U.S. treasury bills	16,922		—		16,922		—
Certificates of deposit	42,337		—		42,337		—
Total	255,698		—		255,698		—
Total cash equivalents and marketable securities	$	300,609	$	44,911	$	255,698	$	—


	December 31, 2021
	Fair Value Measurements
	Total		Level 1		Level 2		Level 3
Assets:
Cash equivalents:
Money market funds	$	119,919	$	119,919	$	—	$	—
Total	119,919		119,919		—		—
Marketable securities:
U.S. government-sponsored entity debt securities	30,614		—		30,614		—
Commercial paper securities	105,757		—		105,757		—
Corporate debt securities	33,682		—		33,682		—
Asset-backed securities	70,701		—		70,701		—
Certificates of deposit	45,091		—		45,091		—
Total	285,845		—		285,845		—
Total cash equivalents and marketable securities	$	405,764	$	119,919	$	285,845	$	—

Cash Equivalents and Marketable Securities

The Company generally classifies its marketable securities as Level 2. Instruments are classified as Level 2 when observable market prices for identical securities that are traded in less active markets are used. When observable market prices for identical securities are not available, such instruments are priced using benchmark curves, benchmarking of like securities, sector groupings, matrix pricing and valuation models. These valuation models are proprietary to the pricing providers or brokers and incorporate a number of inputs, including in approximate order of priority: benchmark yields, reported trades, broker/dealer quotes, issuer spreads, two-sided markets, benchmark securities, bids, offers and reference data including market research publications. For certain security types, additional inputs may be used, or some of the standard inputs may not be applicable. Evaluators may prioritize inputs differently on any given day for any security based on market conditions, and not all inputs listed are available for use in the evaluation process for each security evaluation on any given day.

- Definition

The entire disclosure for the fair value of financial instruments (as defined), including financial assets and financial liabilities (collectively, as defined), and the measurements of those instruments as well as disclosures related to the fair value of non-financial assets and liabilities. Such disclosures about the financial instruments, assets, and liabilities would include: (1) the fair value of the required items together with their carrying amounts (as appropriate); (2) for items for which it is not practicable to estimate fair value, disclosure would include: (a) information pertinent to estimating fair value (including, carrying amount, effective interest rate, and maturity, and (b) the reasons why it is not practicable to estimate fair value; (3) significant concentrations of credit risk including: (a) information about the activity, region, or economic characteristics identifying a concentration, (b) the maximum amount of loss the entity is exposed to based on the gross fair value of the related item, (c) policy for requiring collateral or other security and information as to accessing such collateral or security, and (d) the nature and brief description of such collateral or security; (4) quantitative information about market risks and how such risks are managed; (5) for items measured on both a recurring and nonrecurring basis information regarding the inputs used to develop the fair value measurement; and (6) for items presented in the financial statement for which fair value measurement is elected: (a) information necessary to understand the reasons for the election, (b) discussion of the effect of fair value changes on earnings, (c) a description of [similar groups] items for which the election is made and the relation thereof to the balance sheet, the aggregate carrying value of items included in the balance sheet that are not eligible for the election; (7) all other required (as defined) and desired information.

+ References + Details

CASH EQUIVALENTS AND MARKETABLE SECURITIES

9 Months Ended

Sep. 30, 2022

Investments, Debt and Equity Securities [Abstract]

CASH EQUIVALENTS AND MARKETABLE SECURITIES

The table below summarizes the Company’s cash equivalents and marketable securities (in thousands):


	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses		Estimated Fair Value
September 30, 2022
Assets
Cash equivalents:
Money market funds	$	44,911	$	—	$	—	$	44,911


Total	44,911		—		—		44,911
Marketable securities:
U.S. government-sponsored entity debt securities	23,832		—		(440)		23,392
Commercial paper securities	120,750		11		(375)		120,386
Corporate debt securities	15,050		—		(162)		14,888
Asset-backed securities	38,039		2		(268)		37,773
U.S. treasury bills	16,943		—		(21)		16,922
Certificate of deposits	42,478		5		(146)		42,337
Total	257,092		18		(1,412)		255,698
Total cash equivalents and marketable securities	$	302,003	$	18	$	(1,412)	$	300,609

December 31, 2021
Assets
Cash equivalents:
Money market funds	$	119,919	$	—	$	—	$	119,919
Total	119,919		—		—		119,919
Marketable securities:
U.S. government-sponsored entity debt securities	30,700		1		(87)		30,614
Commercial paper securities	105,792		7		(42)		105,757
Corporate debt securities	33,723		1		(42)		33,682
Asset-backed securities	70,807		1		(107)		70,701
Certificate of deposits	45,116		1		(26)		45,091
Total	286,138		11		(304)		285,845
Total cash equivalents and marketable securities	$	406,057	$	11	$	(304)	$	405,764

The fair value of marketable securities by contractual maturity were as follows (in thousands):


	September 30, 2022		December 31, 2021
Maturing in one year or less	$	218,998	$	197,676
Maturing after one year through five years	36,700		88,169
Total	$	255,698	$	285,845

There were no realized gains and losses on the sales of investments during the three and nine months ended September 30, 2022. Realized gains and losses on the sales of investments were insignificant during the three and nine months ended September 30, 2021.

The Company manages credit risk associated with its investment portfolio through its investment policy, which limits purchases to high-quality issuers and also limits the amount of its portfolio that can be invested in a single issuer. The Company did not record an allowance for credit losses or other impairment charges related to its marketable securities for the three and nine months ended September 30, 2022 and 2021.

The Company had unrealized losses related to its marketable securities for the three and nine months ended September 30, 2022 and 2021. The Company had no material unrealized losses, individually and in the aggregate, for marketable

securities that are in a continuous unrealized loss position for greater than 12 months as of September 30, 2022 and December 31, 2021. Based on the scheduled maturities of its investments, the Company determined that it was more likely than not that it will hold these investments for a period of time sufficient for a recovery of its amortized cost basis. Total unrealized gains for securities with net gains in accumulated other comprehensive income were not material for the three and nine months ended September 30, 2022. The unrealized losses were not attributed to credit risk and were associated with changes in market conditions. The Company periodically reviews its marketable securities for indications of credit losses. The Company considers factors such as the duration, the magnitude and the reason for the decline in value, the potential recovery period, creditworthiness of the issuers of the securities and its intent to sell. For marketable securities, it also considers whether (i) it is more likely than not that the Company will be required to sell the debt securities before recovery of their amortized cost basis, and (ii) the amortized cost basis cannot be recovered as a result of credit losses. No significant facts or circumstances have arisen to indicate that there has been any significant deterioration in the creditworthiness of the issuers of the securities held by the Company. Based on the Company’s review of these securities, including the assessment of the duration and severity of the unrealized losses and the Company’s ability and intent to hold the investments until maturity, the Company determined that no allowance for credit losses related to its marketable securities was required at either September 30, 2022 or December 31, 2021.

BASIC AND DILUTED NET LOSS PER SHARE	9 Months Ended
BASIC AND DILUTED NET LOSS PER SHARE	Sep. 30, 2022
Earnings Per Share [Abstract]
BASIC AND DILUTED NET LOSS PER SHARE	BASIC AND DILUTED NET LOSS PER SHARE Basic net loss per share attributable to Sangamo Therapeutics, Inc. stockholders has been computed by dividing net loss attributable to Sangamo Therapeutics, Inc. stockholders by the weighted-average number of shares of common stock outstanding during the period. Diluted net loss per share attributable to Sangamo Therapeutics, Inc. stockholders is calculated by dividing net loss attributable to Sangamo Therapeutics, Inc. stockholders by the weighted-average number of shares of common stock outstanding during the period, without consideration of any potential dilutive securities, as their effect would be anti-dilutive. The total number of shares subject to stock options and restricted stock units (“RSUs”) outstanding and the employee stock purchase plan (“ESPP”) shares reserved for issuance, which are all anti-dilutive, were excluded from consideration in the calculation of diluted net loss per share attributable to Sangamo Therapeutics, Inc. stockholders. Stock options and RSUs outstanding and ESPP shares reserved for issuance as of September 30, 2022 and 2021 totaled 18,524,016 and 15,838,002, respectively.

MAJOR CUSTOMERS, PARTNERSHIPS AND STRATEGIC ALLIANCES

9 Months Ended

Sep. 30, 2022

Organization, Consolidation and Presentation of Financial Statements [Abstract]

MAJOR CUSTOMERS, PARTNERSHIPS AND STRATEGIC ALLIANCES

Novartis Institutes for BioMedical Research, Inc.

On July 27, 2020, the Company entered into a collaboration and license agreement with Novartis Institutes for BioMedical Research, Inc. (“Novartis”) for the research, development and commercialization of gene regulation therapies to treat three neurodevelopmental disorders. Under the agreement, which was effective upon execution, the Company granted Novartis an exclusive, royalty bearing and worldwide license, under its relevant patents and know-how, to develop, manufacture and commercialize certain of its zinc finger protein (“ZFP”) transcription factors (“ZFP-TFs”) targeted to three undisclosed genes that are associated with certain neurodevelopmental disorders, including autism spectrum disorder and intellectual disability. The Company is performing early research activities over the collaboration period for each gene target and manufacture the ZFP-TFs required for such research, costs of which are funded by Novartis. Novartis is responsible for additional research activities, studies enabling investigational new drug applications (“INDs”), clinical development, regulatory approvals, manufacturing of preclinical, clinical and approved products, and global commercialization. Subject to certain exceptions set forth in the agreement, the Company is prohibited from developing, manufacturing or commercializing any therapeutic product targeting any of the three genes that are the subject of the collaboration. Novartis also has the option to license certain of the Company’s proprietary adeno-associated viruses (“AAVs”) for the sole purpose of developing, manufacturing and commercializing licensed products arising from the collaboration.

Under the agreement, Novartis paid the Company a $75.0 million upfront license fee in August 2020. In addition to this fee and the cost reimbursements for early research activities, the Company is eligible to earn from Novartis up to $420.0 million in development milestones and up to $300.0 million in commercial milestones. The Company is also eligible to earn from Novartis tiered high single-digit to sub-teen double-digit royalties on potential net commercial sales of licensed products arising from the collaboration. These royalty payments will be subject to reduction due to patent expiration, loss of market exclusivity and payments made under certain licenses for third-party intellectual property. The agreement will continue, on a product-by-product and country-by-country basis, until the expiration of the applicable royalty term. Novartis has the right to terminate the agreement, in its entirety or on a target-by-target basis, for any reason after a specified notice period. Each party also has the right to terminate the agreement on account of the other party’s bankruptcy or material, uncured breach.

All payments received under the agreement, when earned, are non-refundable and non-creditable. The transaction price of $95.1 million includes the upfront license fee of $75.0 million and estimated research costs of $20.1 million to be provided over the estimated research period. All clinical or regulatory milestone amounts were considered fully constrained at inception of

the agreement. As part of its evaluation of the constraint, the Company considered numerous factors, including the fact that achievement of the milestones at this time is uncertain and contingent upon future periods when the uncertainty related to the variable consideration is resolved. The Company will re-evaluate the transaction price, including the estimated variable consideration included in the transaction price and all constrained amounts, in each reporting period and as uncertain events are resolved or other changes in circumstances occur.

The Company assessed the agreement with Novartis in accordance with ASC Topic 606 and concluded that Novartis is a customer. The Company has identified a single performance obligation within this arrangement as a license to the technology and ongoing research services. The Company concluded that the license is not discrete as it does not have stand-alone value to Novartis apart from the research services to be performed pursuant to the agreement. As a result, the Company recognizes revenue from the upfront payment based on proportional performance of the ongoing research services through the estimated research period. The estimation of progress towards the satisfaction of performance obligation and project cost is reviewed quarterly and adjusted, as needed, to reflect the Company’s current assumptions regarding the timing of its performance obligation. As of September 30, 2022 and December 31, 2021, the Company had a receivable of $2.0 million and $1.9 million, respectively, and deferred revenue of $17.7 million and $40.9 million, respectively, related to this agreement.

Revenues recognized under the agreement were as follows (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2022		2021		2022		2021
Revenue related to Novartis agreement:
Recognition of upfront license fee	$	7,582	$	9,093	$	23,222	$	22,852
Research services	2,028		2,421		6,211		6,104
Total	$	9,610	$	11,514	$	29,433	$	28,956

The Company paid $1.5 million for financial advisory fees during the year ended December 31, 2020, equal to 2% of $75.0 million received for the upfront license fee related to the collaboration and license agreement with Novartis. The Company recognized $1.5 million as a contract asset as such amount represents a cost of obtaining the agreement. This balance is amortized and included in general and administrative expenses on a systematic basis consistent with the transfer of the services to Novartis in accordance with ASC Topic 340, Other Assets and Deferred Costs (“ASC Topic 340”). The Company amortized $0.2 million and $0.5 million during the three and nine months ended September 30, 2022, respectively, and $0.2 million and $0.5 million during the three and nine months ended September 30, 2021, respectively.

Biogen MA, Inc.

In February 2020, the Company entered into a collaboration and license agreement with Biogen MA, Inc. (“BIMA”) and Biogen International GmbH (together with BIMA, “Biogen”) for the research, development and commercialization of gene regulation therapies for the treatment of neurological diseases. The companies plan to leverage the Company’s proprietary ZFP technology delivered via AAV to modulate expression of key genes involved in neurological diseases. Concurrently with the execution of the collaboration agreement, the Company entered into a stock purchase agreement with BIMA, pursuant to which BIMA agreed to purchase 24,420,157 shares of the Company’s common stock (the “Biogen Shares”), at a price per share of $9.2137, for an aggregate purchase price of approximately $225.0 million.

The collaboration agreement became effective in April 2020 following the termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, and satisfaction of other customary closing conditions, including the payment of $225.0 million for the purchase of the Biogen Shares.

Under the collaboration agreement, Biogen paid the Company an upfront license fee of $125.0 million in May 2020. The Company is also eligible to receive research, development, regulatory and commercial milestone payments that could total up to approximately $2.4 billion if Biogen selects all of the targets allowed under the agreement and all the specified milestones set forth in the agreement are achieved, which includes up to $925.0 million in pre-approval milestone payments and up to $1.5 billion in first commercial sale and other sales-based milestone payments. In addition, the Company is eligible to receive tiered high single-digit to sub-teen royalties on potential net commercial sales of licensed products arising from the collaboration. These royalty payments are subject to reduction due to patent expiration, entry of biosimilar products to the market and payments made under certain licenses for third-party intellectual property.

Under the collaboration agreement, the Company granted to Biogen an exclusive, royalty bearing and worldwide license, under its relevant patents and know-how, to develop, manufacture and commercialize certain ZFP and/or AAV-based products directed to certain neurological disease gene targets selected by Biogen. Biogen has already selected four of these: ST-501 to treat tauopathies, ST-502 to treat synucleinopathies including Parkinson’s disease, a third product candidate targeting DM1, a neuromuscular disease, and a fourth undisclosed neurological disease gene target. Biogen has exclusive rights to nominate up to

seven additional targets over the remaining period of five years from the effective date of the collaboration agreement. For each gene target selected by Biogen, the Company performs early research activities, costs of which are shared by the companies, aimed at the development of the combination of proprietary central nervous system delivery vectors and ZFP-TFs (or potential other ZFP products) targeting therapeutically relevant genes. Biogen has assumed responsibility and costs for the IND-enabling studies, clinical development, related regulatory interactions, and global commercialization. The Company is responsible for manufacturing activities for the initial clinical trials for the first three products of the collaboration and plans to leverage its in-house manufacturing capacity, where appropriate, which is currently in development. Biogen is responsible for manufacturing activities beyond the first clinical trial for each of the first three products. The Company’s research activities for any targets will be performed over the period not to exceed seven years from the effective date of the agreement (i.e., through April 2027). Subject to certain exceptions set forth in the collaboration agreement, the Company is prohibited from developing, manufacturing or commercializing any therapeutic product directed to the targets selected by Biogen.

The collaboration agreement continues on a product-by-product and country-by-country basis until the expiration of all applicable royalty terms. Biogen has the right to terminate the collaboration agreement, in its entirety or on a target-by-target basis, for any reason after a specified notice period, and also has the right to replace up to ten targets. Each party has the right to terminate this agreement on account of the other party’s bankruptcy or material, uncured breach. In addition, the Company may terminate the collaboration agreement if Biogen challenges any patents licensed by the Company to Biogen.

Pursuant to the terms of the stock purchase agreement, Biogen has agreed not to, without the Company’s prior written consent and subject to specified conditions and exceptions, directly or indirectly acquire shares of the Company’s outstanding common stock, seek or propose a tender or exchange offer or merger between the parties, solicit proxies or consents with respect to any matter, or undertake other specified actions related to the potential acquisition of additional equity interests in the Company. Such standstill restrictions expire on the earlier of the three-year anniversary of the effectiveness of the collaboration agreement and the date that Biogen beneficially owns less than 5% of the Company’s common stock.

The Company assessed the collaboration agreement with Biogen in accordance with ASC Topic 606 and concluded that Biogen is a customer. The transaction price of $204.6 million includes the upfront license fee of $125.0 million and the excess consideration from the stock purchase of $79.6 million, which represents the difference between the $225.0 million received for the purchase of the Biogen Shares and the $145.4 million estimated fair value of the equity issued. The equity issued to Biogen was valued using an option pricing model to reflect certain holding period restrictions. None of the target selection fees and clinical or regulatory milestones have been included in the transaction price, as all such amounts are fully constrained. As part of its evaluation of the constraint, the Company considered numerous factors, including the fact that nomination of additional targets and achievement of the milestones at this time is uncertain and contingent upon future periods when the uncertainty related to the variable consideration is resolved. The Company will re-evaluate the transaction price as uncertain events are resolved or other changes in circumstances occur.

The Company has identified a single performance obligation within the Biogen collaboration agreement, which is a stand-ready obligation consisting of a series of distinct days of research services, during which Biogen obtains access to the Company’s license and research resources. Revenue from the upfront license fee relates to access to the license and Company’s obligation to stand-ready to perform such research services corresponding to the targets selected by Biogen. As a result of this obligation to perform research services when and if requested throughout the duration of the contract, the upfront license fee and the excess consideration from the stock purchase will be recognized over time on a straight-line basis consistent with the resources expected to be dedicated to providing the research services through April 2027, the estimated period of the obligation. The estimated period of performance is reviewed quarterly and adjusted, as needed, to reflect the Company’s current assumptions regarding the timing of its deliverable. Revenue from the reimbursement by Biogen of shared costs of early research activities performed by Sangamo is recognized as the research services are performed. As of September 30, 2022 and December 31, 2021, the Company had a receivable of $1.7 million and $2.8 million, respectively, and deferred revenue of $132.1 million and $154.0 million, respectively, related to this agreement.

Revenues recognized under the agreement were as follows (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2022		2021		2022		2021
Revenue related to Biogen agreement:
Recognition of license and stand-ready fee	$	7,306	$	7,306	$	21,918	$	21,918
Research services	2,107		3,661		8,740		10,266
Total	$	9,413	$	10,967	$	30,658	$	32,184

The Company paid $7.0 million for financial advisory fees during the year ended December 31, 2020, equal to 2% of $225.0 million received for the sale of shares and 2% of $125.0 million received for the upfront fee. The fees incurred related to

both the collaboration agreement with Biogen and the stock purchase agreement for the sale of shares. The Company believes that the allocation of fees on a relative fair value basis between the two agreements is reasonable. The Company recognized $4.1 million, which represents 2% of the transaction price of $204.6 million, as a contract asset. This balance is amortized and included in general and administrative expenses on a systematic basis consistent with the transfer of the services to Biogen in accordance with ASC Topic 340. The Company amortized $0.1 million and $0.4 million during the three and nine months ended September 30, 2022, respectively, and $0.1 million and $0.4 million during the three and nine months ended September 30, 2021, respectively. The Company recognized $2.9 million, which represented 2% of the $145.4 million estimated fair value of the equity issued, as a share issuance cost and recorded this amount in equity as a reduction in net proceeds during the year ended December 31, 2020.

Kite Pharma, Inc.

In February 2018, the Company entered into a global collaboration and license agreement with Kite Pharma, Inc. (“Kite”), a Gilead Sciences, Inc. subsidiary, which became effective on April 5, 2018 (“Effective Date”), and was amended and restated in September 2019, for the research, development, and commercialization of potential engineered cell therapies for cancer. In this collaboration, Sangamo is working together with Kite on a research program under which the companies are designing zinc finger nucleases (“ZFNs”) and viral vectors to disrupt and insert certain genes in T-cells and natural killer cells (“NK-cells”) including the insertion of genes that encode chimeric antigen receptors (“CARs”), T-cell receptors (“TCRs”), and NK-cell receptors (“NKRs”) directed to mutually agreed targets. Kite is responsible for all clinical development, manufacturing and commercialization of any resulting products.

Subject to the terms of this agreement, the Company granted Kite an exclusive, royalty-bearing, worldwide sublicensable license under the Company’s relevant patents and know-how to develop, manufacture and commercialize, for the purpose of treating cancer, specific cell therapy products that may result from the research program and that are engineered ex vivo using selected ZFNs and viral vectors developed under the research program to express CARs, TCRs or NKRs directed to candidate targets.

During the research program term and subject to certain exceptions, the Company is prohibited from researching, developing, manufacturing and commercializing, for the purpose of treating cancer, any cell therapy product that, as a result of ex vivo genome editing, expresses a CAR, TCR or NKR that is directed to a target expressed on or in a human cancer cell. After the research program term concludes and subject to certain exceptions, the Company will be prohibited from developing, manufacturing and commercializing, for the purpose of treating cancer, any cell therapy product that, as a result of ex vivo genome editing, expresses a CAR, TCR or NKR that is directed to a candidate target.

Following the Effective Date, the Company received a $150.0 million upfront payment from Kite. In addition, Kite reimburses the Company’s direct costs to conduct the joint research program. Sangamo is also eligible to receive contingent development- and sales-based milestone payments that could total up to $3.0 billion if all of the specified milestones set forth in this agreement are achieved. Of this amount, approximately $1.3 billion relates to the achievement of specified research, clinical development, regulatory and first commercial sale milestones, and approximately $1.8 billion relates to the achievement of specified sales-based milestones if annual worldwide net sales of licensed products reach specified levels. Each development- and sales-based milestone payment is payable (i) only once for each licensed product, regardless of the number of times that the associated milestone event is achieved by such licensed product, and (ii) only for the first 10 times that the associated milestone event is achieved regardless of the number of licensed products that may achieve such milestone event. In addition, the Company is entitled to receive escalating, tiered royalty payments with a percentage in the single digits based on future annual worldwide net sales of licensed products. These royalty payments are subject to reduction due to patent expiration, entry of biosimilar products to the market and payments made under certain licenses for third-party intellectual property.

The initial research term in the agreement is six years from the Effective Date. Kite has an option to extend the research term for up to two additional one-year periods for a separate upfront fee of $10.0 million per year. All contingent payments under the agreement, when earned, will be non-refundable and non-creditable. In connection with the amendment and restatement of the agreement in September 2019, the Company entered into a new research plan with Kite, with estimated reimbursable service cost of approximately $3.4 million, which is included in the total transaction price. The Company concluded the total transaction price under this agreement is $189.3 million and includes the upfront license fee of $150.0 million and $39.3 million estimated reimbursable service costs for identified research projects over the estimated performance period. Further, the Company concluded the estimated fees for the presumed exercise of the research term extension options and all milestone amounts are fully constrained. As part of its evaluation of the constraint, the Company considered numerous factors, including the fact that achievement of the milestones at this time is uncertain and contingent upon future events which are uncertain at this time. The Company will re-evaluate the transaction price including the estimated variable consideration included in the transaction price and all constrained amounts in each reporting period and as uncertain events are resolved or other changes in circumstances occur. None of the development and sales-based milestone payments have been included in the transaction price.

The Company assessed the agreement with Kite in accordance with ASC Topic 606 and concluded that Kite is a customer. Kite has the right to terminate this agreement in its entirety or on a per licensed product or per candidate target basis for any reason after a specified notice period. Each party has the right to terminate this agreement on account of the other party’s bankruptcy or material, uncured breach.

The Company has identified the primary performance obligations within the Kite agreement as: (1) a license to the technology along with the stand-ready obligation to perform research services, and (2) the ongoing research services. Revenue from the upfront license fee relates to access to the license and Company’s obligation to stand-ready to perform such research services as additional targets are selected by Kite. As a result of this obligation to perform research services when and if requested throughout the duration of the contract, the fee for the license and the stand-ready obligation will be recognized over time on a straight-line basis through April 2024, the estimated period of the stand-ready obligation. Revenue from the reimbursable costs related to the integrated service deliverable is recognized as the research services are performed. Related costs and expenses under these arrangements have historically approximated the revenues recognized. The estimated period of performance and project cost is reviewed quarterly and adjusted, as needed, to reflect the Company’s current assumptions regarding the timing of its deliverables. As of September 30, 2022 and December 31, 2021, the Company had a receivable of $0.6 million and $0.1 million, respectively, and deferred revenue of $37.8 million and $56.5 million, respectively, related to this agreement.

Revenues recognized under the agreement were as follows (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2022		2021		2022		2021
Revenue related to Kite agreement:
Recognition of license and stand-ready fee	$	6,296	$	6,296	$	18,682	$	18,682
Research services	619		113		875		339
Total	$	6,915	$	6,409	$	19,557	$	19,021

Sanofi S.A.

In January 2014, the Company entered into an exclusive worldwide collaboration and license agreement (“2014 Collaboration Agreement”) to develop therapeutics for hemoglobinopathies, focused on beta thalassemia and sickle cell disease (“SCD”). The 2014 Collaboration Agreement was originally signed with BIMA, who subsequently assigned it to Bioverativ Inc., which was later acquired by Sanofi. Under the 2014 Collaboration Agreement, the Company was originally jointly conducting two research programs: a beta thalassemia program, which was discontinued in the third quarter of 2021, and the SCD program, which resulted in the development of SAR445136 (now known as BIVV003), a ZFN, gene-edited cell therapy product candidate for the treatment of SCD, which remains active. In December 2021, Sanofi notified the Company of its termination for convenience, effective June 28, 2022 (the “Termination Date”), of the 2014 Collaboration Agreement. A termination and transition agreement (the “Termination and Transition Agreement”) was executed by the parties on September 6, 2022.

In the SCD program, the Company and Sanofi were jointly responsible for research and development activities prior to filing of an IND, but Sanofi was responsible for subsequent worldwide clinical development, manufacturing and commercialization of licensed products developed under the agreement. Subject to the terms of the agreement, the Company had granted Sanofi an exclusive, royalty-bearing license, with the right to grant sublicenses, to use certain ZFP and other technology controlled by the Company for the purpose of researching, developing, manufacturing and commercializing licensed products developed under the agreement. The Company had also granted Sanofi a non-exclusive worldwide, royalty-free fully paid license with the right to grant sublicenses, under the Company’s interest in certain other intellectual property developed pursuant to the agreement. During the term of the agreement, the Company was not permitted to research, develop, manufacture or commercialize, outside of the agreement, certain gene therapy products that target genes relevant to the licensed products.

Under the agreement, the Company received an upfront license fee of $20.0 million and was eligible to receive additional payments upon the achievement of specified clinical development, regulatory and sales milestones, as well as royalty payments for each licensed product based on net sales of such product. Sanofi was also to reimburse Sangamo for agreed upon costs incurred in connection with research and development activities conducted by Sangamo. Through the Termination Date, a total of $13.5 million has been received based upon achievement of clinical development milestones. No products have been approved and therefore no royalty fees have been or will be earned under the 2014 Collaboration Agreement.

In its termination notice to the Company, Sanofi indicated that its termination relates to Sanofi’s change in strategic direction to focus on allogeneic universal genomic medicine approaches rather than autologous personalized cell therapies. As of the Termination Date, the 2014 Collaboration Agreement was terminated in its entirety and following the Termination Date, the Company will not be entitled to receive any further milestone payments or royalties from Sanofi. As of the Termination Date, Sanofi has no further obligations under the 2014 Collaboration Agreement to develop or to fund the development of any

collaboration research programs under the 2014 Collaboration Agreement. The licenses granted to Sanofi under the 2014 Collaboration Agreement have been terminated, and the license rights have reverted to the Company.

As part of the Termination and Transition Agreement, Sanofi granted to the Company exclusive, worldwide, fully paid, royalty-free, perpetual, irrevocable licenses, with the right to grant sublicenses through multiple tiers, to certain of its intellectual property, to develop, manufacture, use, sell and otherwise commercialize BIVV003, the product candidate in development under the SCD program. The Company has agreed to take on responsibilities for all clinical trials related to BIVV003, including completion of the ongoing clinical trial and the related long-term follow-up study. The Company also assumed all regulatory responsibilities related to BIVV003. Sanofi has agreed to provide post-transition assistance to the Company for up to 90 days after the Termination Date, and transferred or assigned to the Company all documentation, materials, contracts with third parties, and the right to use certain Sanofi-owned equipment related to BIVV003.

Sanofi has also agreed to reimburse the Company for the costs of conducting the ongoing clinical trial of BIVV003 and the costs of the long-term follow-up study through December 31, 2023, up to $7.0 million. In addition, should the Company elect not to continue the development of BIVV003 past December 31, 2023, Sanofi will become obligated to reimburse the Company for the costs of the long-term follow-up study incurred after 2023, up to $5.3 million. Sanofi’s reimbursement obligations will terminate upon certain triggering events, including the Company’s entering into a contract with a third party for collaboration, partnership, sale, licensing, or divestiture of BIVV003, or if the FDA permits early closure of the clinical trial and/or the long-term follow-up study.

The Company assessed the 2014 Collaboration Agreement with Sanofi in accordance with ASC Topic 606 and concluded that Sanofi was a customer, under that arrangement. The Company identified the performance obligation within this arrangement as a license to the technology combined with ongoing research services activities. The Company concluded that the license was not distinct as it did not have value to Sanofi without the research services. As a result, the Company recognized revenue from the upfront payment and the milestones based on progress of performance of the ongoing research services. The estimation of progress towards the satisfaction of the performance obligation and project cost was reviewed quarterly and adjusted, as needed, to reflect the Company’s then-current assumptions regarding the timing of its deliverables. Related costs and expenses under these arrangements have historically approximated the revenues recognized. Sanofi’s December 2021 notice of termination of the 2014 Collaboration Agreement represented a modification that reduced the expected scope of the Company’s services and the estimated transaction price and shortened the remaining performance timeline. Consistent with this change, all services under the 2014 Collaboration Agreement were completed by June 28, 2022, and all amounts ultimately included in the transaction price were recognized by such date. The final transaction price of $96.3 million includes the upfront license fee of $20.0 million, two milestone payments in the aggregate amount of $13.5 million and reimbursement of research costs of $62.8 million. As of September 30, 2022 and December 31, 2021, the Company had a receivable of zero and $0.6 million, respectively, related to the 2014 Collaboration Agreement. Deferred revenue related to the 2014 Collaboration Agreement was zero and $1.1 million as of September 30, 2022 and December 31, 2021, respectively.

The Company concluded that Sanofi is not a customer under the Termination and Transition Agreement as Sanofi is not entitled to receive and cannot use the results of the ongoing clinical trial or the long-term follow-up study. This relationship is also not a collaboration in the scope of ASC Topic 808, Collaborative Arrangements. The Company concluded that the assets acquired from Sanofi do not represent a business, as substantially all of their value is concentrated in the acquired or re-acquired licenses to intellectual property. The Company has no obligation to repay Sanofi for its ongoing funding of the clinical trial or long-term follow-up study costs. Therefore, the Company will recognize Sanofi reimbursements as reductions of its research and development expense. During the three and nine months ended September 30, 2022, the Company decreased its research and development expense by $0.9 million, which is included within prepaid expenses and other current assets on the Company’s Condensed Consolidated Balance Sheet as of September 30, 2022.

Revenues recognized under the 2014 Collaboration Agreement were as follows (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2022		2021		2022		2021
Revenue related to Sanofi agreement:
Recognition of upfront fee	$	—	$	(1,125)	$	677	$	(692)
Research services	—		1,125		2,126		2,417
Milestone achievement	—		(759)		457		(467)
Total	$	—	$	(759)	$	3,260	$	1,258

In September 2021, the Company recorded an adjustment to revenue related to a change in estimate in connection with the collaboration agreement with Sanofi. This adjustment was a result of an increase in the project costs for its beta thalassemia program and the increase in project scope and the corresponding costs for its SCD program, both of which resulted in a decrease

in the measure of proportional cumulative performance. This adjustment decreased revenue by $2.5 million, increased net loss by $2.5 million and increased the Company's basic and diluted net loss per share by $0.02 for the three and nine months ended September 30, 2021.

Pfizer Inc.

Giroctocogene Fitelparvovec Global Collaboration and License Agreement

In May 2017, the Company entered into an exclusive global collaboration and license agreement with Pfizer Inc. (“Pfizer”), pursuant to which it established a collaboration for the research, development and commercialization of giroctocogene fitelparvovec, its gene therapy product candidate for hemophilia A, and closely related products.

Under this agreement, the Company is responsible for conducting the Phase 1/2 clinical trial and for certain manufacturing activities for giroctocogene fitelparvovec, while Pfizer is responsible for subsequent worldwide development, manufacturing, marketing and commercialization of giroctocogene fitelparvovec. Sangamo may also collaborate in the research and development of additional AAV-based gene therapy products for hemophilia A.

Subject to the terms of the agreement, the Company granted Pfizer an exclusive worldwide royalty-bearing license, with the right to grant sublicenses, to use certain technology controlled by the Company for the purpose of developing, manufacturing and commercializing giroctocogene fitelparvovec and related products. Pfizer granted the Company a non-exclusive, worldwide, royalty-free, fully paid license, with the right to grant sublicenses, to use certain manufacturing technology developed under the agreement and controlled by Pfizer to manufacture the Company’s products that utilize the AAV delivery system. During a specified period, neither the Company nor Pfizer is permitted to clinically develop or commercialize, outside of the collaboration, certain AAV-based gene therapy products for hemophilia A.

Unless earlier terminated, the agreement has a term that continues on a per product and per country basis until the later of (i) the expiration of patent claims that cover the product in a country, (ii) the expiration of regulatory exclusivity for a product in a country, and (iii) fifteen years after the first commercial sale of a product in a country. Pfizer has the right to terminate the agreement without cause in its entirety or on a per product or per country basis. The agreement may also be terminated by either party based on an uncured material breach by the other party or the bankruptcy of the other party. Upon termination for any reason, the license granted by the Company to Pfizer to develop, manufacture and commercialize giroctocogene fitelparvovec and related products will automatically terminate. Upon termination by the Company for cause or by Pfizer in any country or countries, Pfizer will automatically grant the Company an exclusive, royalty-bearing license under certain technology controlled by Pfizer to develop, manufacture and commercialize giroctocogene fitelparvovec in the terminated country or countries.

Upon execution of the agreement, the Company received an upfront fee of $70.0 million and is eligible to receive up to $208.5 million in payments upon the achievement of specified clinical development, intellectual property and regulatory milestones and up to $266.5 million in payments upon first commercial sale milestones for giroctocogene fitelparvovec and potentially other products. The total amount of potential clinical development, intellectual property, regulatory and first commercial sale milestone payments, assuming the achievement of all specified milestones in the agreement, is up to $475.0 million, which includes up to $300.0 million for giroctocogene fitelparvovec and up to $175.0 million for other products that may be developed under the agreement, subject to reduction on account of payments made under certain licenses for third-party intellectual property. In addition, Pfizer agreed to pay the Company royalties for each potential licensed product developed under the agreement that are an escalating tiered, double-digit percentage of the annual net sales of such product and are subject to reduction due to patent expiration, entry of biosimilar products to the market and payment made under certain licenses for third-party intellectual property. To date, two milestones of $55.0 million in aggregate have been achieved and paid, however no products have been approved and therefore no royalty fees have been earned under the agreement.

The Company assessed the agreement with Pfizer in accordance with ASC Topic 606 and concluded that Pfizer is a customer. The total transaction price under this agreement was $134.0 million, which represented the upfront fee and research services fees of $79.0 million and fees related to two achieved milestones in an aggregate amount of $55.0 million. Sangamo was responsible for internal and external research costs as part of the upfront fee and had the ability to request additional reimbursement from Pfizer if certain conditions were met. None of the constrained clinical or regulatory milestones were included in the transaction price. As part of its evaluation of the constraint, the Company considered numerous factors, including the fact that achievement of the milestones at the time was uncertain and contingent upon future periods when the uncertainty related to the variable consideration is resolved. The Company will re-evaluate the transaction price, including its estimated variable consideration included in the transaction price and all constrained amounts, in each reporting period and as uncertain events are resolved or other changes in circumstances occur.

The Company has identified the performance obligations within the agreement as a license to the technology and ongoing research services. The Company concluded that the license is not discrete as it does not have stand-alone value to Pfizer apart from the research services to be performed by the Company pursuant to the agreement. As a result, the Company recognized

revenue from the upfront payment based on proportional performance of the ongoing research services through 2020, the period during which the Company performed research services. The estimation of progress towards the satisfaction of its performance obligation and project cost was reviewed quarterly and adjusted, as needed, to reflect the Company’s assumptions regarding the timing of its deliverables.

In December 2020, the Company satisfied the deliverables and research services responsibilities within the arrangement. As a result, the Company recognized the remaining deferred revenue from the upfront payment in December 2020 and no revenues have been recognized during the three and nine months ended September 30, 2022 and 2021.

C9ORF72 Research Collaboration and License Agreement

In December 2017, the Company entered into a separate exclusive, global collaboration and license agreement with Pfizer for the development and commercialization of potential gene therapy products that use ZFP-TFs to treat amyotrophic lateral sclerosis and frontotemporal lobar degeneration linked to mutations of the C9ORF72 gene. Pursuant to this agreement, the Company agreed to work with Pfizer on a research program to identify, characterize and preclinically develop ZFP-TFs that bind to and specifically reduce expression of the mutant form of the C9ORF72 gene.

Subject to the terms of this agreement, the Company granted Pfizer an exclusive, royalty-bearing, worldwide license under the Company’s relevant patents and know-how to develop, manufacture and commercialize gene therapy products that use resulting ZFP-TFs that satisfy pre-agreed criteria. During a specified period, neither the Company nor Pfizer will be permitted to research, develop, manufacture or commercialize outside of the collaboration any ZFPs that specifically bind to the C9ORF72 gene.

Unless earlier terminated, the agreement has a term that continues on a per licensed product and per country basis until the later of (i) the expiration of patent claims that cover the licensed product in a country, (ii) the expiration of regulatory exclusivity for a licensed product in a country, and (iii) 15 years after the first commercial sale of a licensed product in a major market country. Pfizer also has the right to terminate the agreement without cause in its entirety or on a per product or per country basis. The agreement may also be terminated by either party based on an uncured material breach by the other party or the bankruptcy of the other party. The agreement will also terminate if the Company is unable to identify any lead candidates for development within a specified period of time or if Pfizer elects not to advance a lead candidate beyond a certain development milestone within a specified period of time. Upon termination for any reason, the license granted by the Company to Pfizer to develop, manufacture and commercialize licensed products under the agreement will automatically terminate. Upon termination by the Company for cause or by Pfizer without cause for any licensed product or licensed products in any country or countries, the Company will have the right to negotiate with Pfizer to obtain a non-exclusive, royalty-bearing license under certain technology controlled by Pfizer to develop, manufacture and commercialize the licensed product or licensed products in the terminated country or countries.

Following termination by the Company for Pfizer’s material breach, Pfizer will not be permitted to research, develop, manufacture or commercialize ZFPs that specifically bind to the C9ORF72 gene for a period of time. Following termination by Pfizer for the Company’s material breach, the Company will not be permitted to research, develop, manufacture or commercialize ZFPs that specifically bind to the C9ORF72 gene for a period of time.

The Company received a $12.0 million upfront payment from Pfizer and is eligible to receive up to $60.0 million in development milestone payments from Pfizer contingent on the achievement of specified preclinical development, clinical development and first commercial sale milestones, and up to $90.0 million in commercial milestone payments if annual worldwide net sales of the licensed products reach specified levels. In addition, Pfizer will pay the Company royalties based on an escalating tiered, mid- to high-single digit percentage of the annual worldwide net sales of the licensed products. These royalty payments are subject to reduction due to patent expiration, entry of biosimilar products to the market and payments made under certain licenses for third-party intellectual property. Each party will be responsible for the cost of its performance of the research program. Pfizer will be operationally and financially responsible for subsequent development, manufacturing and commercialization of the licensed products. To date, a milestone of $5.0 million has been achieved and paid, however no products have been approved and therefore no royalty fees have been earned under the C9ORF72 Pfizer agreement.

The Company assessed the agreement with Pfizer in accordance with ASC Topic 606 and concluded that Pfizer was a customer. The Company concluded the total transaction price under this agreement was $17.0 million, which represented the upfront fees of $12.0 million and fees related to achievement of one milestone in the amount of $5.0 million. None of the constrained clinical or regulatory milestones were included in the transaction price. As part of its evaluation of the constraint, the Company considered numerous factors, including the fact that achievement of the milestones at the time was uncertain and contingent upon future periods when the uncertainty related to the variable consideration is resolved. The Company will evaluate the transaction price, including its estimated variable consideration included in the transaction price and all constrained amounts, in each reporting period and as uncertain events are resolved or other changes in circumstances occur.

The Company had identified the performance obligations within this agreement as a license to the technology and ongoing research services. The Company concluded that the license is not discrete as it does not have stand-alone value to Pfizer apart from the services to be performed by the Company pursuant to the agreement. As a result, the Company recognized revenue from the upfront payment based on proportional performance of the ongoing research services through 2020, the period the Company performed research services.The Company satisfied the deliverables and research services responsibilities within the arrangement in September 2020, and as a result, earned a $5.0 million milestone, which the Company recognized on a cumulative basis during the year ended December 31, 2020. In addition, the Company recognized the remaining deferred revenue from the upfront payment in September 2020 and no revenues have been recognized during the three and nine months ended September 30, 2022 and 2021.

STOCK-BASED COMPENSATION

9 Months Ended

Sep. 30, 2022

Share-Based Payment Arrangement [Abstract]

STOCK-BASED COMPENSATION

STOCK-BASED COMPENSATIONThe following table shows total stock-based compensation expense included in the Condensed Consolidated Statements of Operations (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2022		2021		2022		2021
Research and development	$	4,395	$	4,935	$	13,656	$	14,645
General and administrative	3,398		2,938		9,746		10,235
Total stock-based compensation expense	$	7,793	$	7,873	$	23,402	$	24,880

STOCKHOLDERS' EQUITY	9 Months Ended
STOCKHOLDERS' EQUITY	Sep. 30, 2022
Equity [Abstract]
STOCKHOLDERS' EQUITY	STOCKHOLDERS’ EQUITY At-the-Market Offering Agreement In August 2020, the Company entered into an Open Market Sale Agreement℠ with Jefferies LLC (“Jefferies”) with respect to an at-the-market offering program under which the Company may offer and sell, from time to time at its sole discretion, shares of the Company’s common stock having an aggregate offering price of up to $150.0 million through Jefferies as the Company’s sales agent or principal. The Company is not obligated to sell any shares under the sales agreement. During the three and nine months ended September 30, 2022, the Company sold 8,483,104 and 14,711,770 shares of its common stock for net proceeds of approximately $42.2 million and $66.4 million, respectively, of which approximately $0.6 million was received in October 2022 and recorded within prepaid expenses and other current assets on the Company’s Condensed Consolidated Balance Sheet as of September 30, 2022. During the three and nine months ended September 30, 2021, the Company sold 202,705 and 2,007,932 shares of its common stock for net proceeds of approximately $2.4 million, and $27.1 million, respectively.

- Definition

The entire disclosure for shareholders' equity comprised of portions attributable to the parent entity and noncontrolling interest, including other comprehensive income. Includes, but is not limited to, balances of common stock, preferred stock, additional paid-in capital, other capital and retained earnings, accumulated balance for each classification of other comprehensive income and amount of comprehensive income.

+ References

Reference 1: http://fasb.org/us-gaap/role/ref/legacyRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 505
-URI https://asc.fasb.org/topic&trid=2208762

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 505
-SubTopic 10
-Section 50
-Paragraph 13
-Subparagraph (b)
-URI https://asc.fasb.org/extlink&oid=126973232&loc=SL123496158-112644

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 505
-SubTopic 10
-Section 50
-Paragraph 13
-URI https://asc.fasb.org/extlink&oid=126973232&loc=SL123496158-112644

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 505
-SubTopic 10
-Section 50
-Paragraph 14
-Subparagraph (c)
-URI https://asc.fasb.org/extlink&oid=126973232&loc=SL123496171-112644

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 505
-SubTopic 10
-Section 50
-Paragraph 14
-Subparagraph (b)
-URI https://asc.fasb.org/extlink&oid=126973232&loc=SL123496171-112644

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 505
-SubTopic 10
-Section S99
-Paragraph 1
-Subparagraph (SX 210.3-04)
-URI https://asc.fasb.org/extlink&oid=120397183&loc=d3e187085-122770

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+ Details

INCOME TAXES	9 Months Ended
INCOME TAXES	Sep. 30, 2022
Income Tax Disclosure [Abstract]
INCOME TAXES	INCOME TAXES The Company’s provision for income taxes for interim periods is determined using an estimate of its annual effective tax rate, adjusted for discrete items, if any, that arise during the period. Each quarter, the Company updates its estimate of the annual effective tax rate, and if the estimated annual effective tax rate changes, the Company makes a cumulative adjustment in such period. During the three and nine months ended September 30, 2022, the Company recorded income tax expense of $0.1 million and $0.2 million, respectively. During the three and nine months ended September 30, 2021, the Company recorded income tax expense of $0.1 million and $0.4 million, respectively. The Company continues to maintain a full valuation allowance on its U.S. federal and state net deferred tax assets and on the Sangamo France net deferred tax assets, as the Company believes it is not more likely than not that these benefits will be realized. The tax expense for the three and nine months ended September 30, 2022 was primarily due to foreign income tax expense. The Coronavirus Aid, Relief, and Economic Security Act, enacted on March 27, 2020, provided an Employee Retention Credit (“ERC”), which is a refundable payroll tax credit that encouraged businesses to keep employees on the payroll during the COVID-19 pandemic. Eligible employers could qualify for up to $5,000 of credit for each employee based on certain wages paid after March 12, 2020 and before January 1, 2021 and extended to September 30, 2021. Based on the Company’s evaluation of this provision and the significant pandemic-related impacts on the operations, the Company recognized zero and $3.0 million of ERC for the three and nine months ended September 30, 2022, respectively, within interest and other income, net in Condensed Consolidated Statement of Operations upon filing for the refund in January and April 2022. No refund has been received during the three and nine months ended September 30, 2022 and the amount has been recorded within prepaid expenses and other current assets on the Company’s Condensed Consolidated Balance Sheet as of September 30, 2022. On August 16, 2022, the Inflation Reduction Act of 2022 was signed into law, with tax provisions primarily focused on implementing a 15% minimum tax on global adjusted financial statement income and a 1% excise tax on share repurchases. The Inflation Reduction Act of 2022 will become effective beginning in 2023. Given its recent pronouncement, it is unclear at this time what, if any, impact the Inflation Reduction Act of 2022 will have on the Company's tax rate and financial results. Currently, the Company does not anticipate any material impact on its consolidated audited financial statements and will continue to evaluate its impact as further information becomes available.

- Definition

The entire disclosure for income taxes. Disclosures may include net deferred tax liability or asset recognized in an enterprise's statement of financial position, net change during the year in the total valuation allowance, approximate tax effect of each type of temporary difference and carryforward that gives rise to a significant portion of deferred tax liabilities and deferred tax assets, utilization of a tax carryback, and tax uncertainties information.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
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+ Details

ORGANIZATION, BASIS OF PRESENTATION, AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - (Policies)

9 Months Ended

Sep. 30, 2022

Accounting Policies [Abstract]

Basis of Presentation

Use of Estimates

making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Actual results could differ from those estimates.

Revenue Recognition

Revenues from major collaboration agreements and research activity grants as a percentage of total revenues were as follows:


	Three Months Ended September 30,				Nine Months Ended September 30,
	2022		2021		2022		2021
Novartis Institutes for BioMedical Research, Inc.	36	%	40	%	35	%	35	%
Biogen MA, Inc.	36	%	38	%	36	%	39	%
Kite Pharma, Inc.	26	%	22	%	23	%	23	%
Sanofi S.A.	—	%	(3)	%	4	%	2	%

Cash, Cash Equivalents and Restricted Cash

Cash, Cash Equivalents, and Restricted Cash

Recently Adopted Accounting Pronouncements

None.

Fair Value Measurements

FAIR VALUE MEASUREMENTS

Level 1: Unadjusted quoted prices in active markets that are accessible at the measurement date for identical, unrestricted assets or liabilities;

Level 2: Quoted prices in markets that are not active or inputs which are observable, either directly or indirectly, for substantially the full term of the asset or liability; and

Level 3: Prices or valuation techniques that require inputs that are both significant to the fair value measurements and unobservable (i.e., supported by little or no market activity).

- Definition

Disclosure of accounting policy for revenue from contract with customer.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
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+ Details

ORGANIZATION, BASIS OF PRESENTATION, AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - (Tables)

9 Months Ended

Sep. 30, 2022

Accounting Policies [Abstract]

Schedule of Revenue from Strategic Partnering Collaboration Agreements and Research Activity Grants as a Percentage of Total Revenues

Revenues from major collaboration agreements and research activity grants as a percentage of total revenues were as follows:


	Three Months Ended September 30,				Nine Months Ended September 30,
	2022		2021		2022		2021
Novartis Institutes for BioMedical Research, Inc.	36	%	40	%	35	%	35	%
Biogen MA, Inc.	36	%	38	%	36	%	39	%
Kite Pharma, Inc.	26	%	22	%	23	%	23	%
Sanofi S.A.	—	%	(3)	%	4	%	2	%

Schedule of Cash and Cash Equivalents


	September 30, 2022		December 31, 2021		September 30, 2021		December 31, 2020
Cash and cash equivalents	$	94,570	$	178,872	$	176,954	$	131,329
Non-current restricted cash	1,500		1,500		1,500		1,500
Cash, cash equivalents, and restricted cash as reported within the accompanying Condensed Consolidated Statements of Cash Flows	$	96,070	$	180,372	$	178,454	$	132,829

Restrictions on Cash and Cash Equivalents


	September 30, 2022		December 31, 2021		September 30, 2021		December 31, 2020
Cash and cash equivalents	$	94,570	$	178,872	$	176,954	$	131,329
Non-current restricted cash	1,500		1,500		1,500		1,500
Cash, cash equivalents, and restricted cash as reported within the accompanying Condensed Consolidated Statements of Cash Flows	$	96,070	$	180,372	$	178,454	$	132,829

FAIR VALUE MEASUREMENTS - (Tables)

9 Months Ended

Sep. 30, 2022

Fair Value Disclosures [Abstract]

Summary of Fair Value Measurements of Cash Equivalents and Marketable Securities

The fair value measurements of the Company’s cash equivalents and marketable securities are identified at the following levels within the fair value hierarchy (in thousands):


	September 30, 2022
	Fair Value Measurements
	Total		Level 1		Level 2		Level 3
Assets:
Cash equivalents:
Money market funds	$	44,911	$	44,911	$	—	$	—


Total	44,911		44,911		—		—
Marketable securities:
U.S. government-sponsored entity debt securities	23,392		—		23,392		—
Commercial paper securities	120,386		—		120,386		—
Corporate debt securities	14,888		—		14,888		—
Asset-backed securities	37,773		—		37,773		—
U.S. treasury bills	16,922		—		16,922		—
Certificates of deposit	42,337		—		42,337		—
Total	255,698		—		255,698		—
Total cash equivalents and marketable securities	$	300,609	$	44,911	$	255,698	$	—


	December 31, 2021
	Fair Value Measurements
	Total		Level 1		Level 2		Level 3
Assets:
Cash equivalents:
Money market funds	$	119,919	$	119,919	$	—	$	—
Total	119,919		119,919		—		—
Marketable securities:
U.S. government-sponsored entity debt securities	30,614		—		30,614		—
Commercial paper securities	105,757		—		105,757		—
Corporate debt securities	33,682		—		33,682		—
Asset-backed securities	70,701		—		70,701		—
Certificates of deposit	45,091		—		45,091		—
Total	285,845		—		285,845		—
Total cash equivalents and marketable securities	$	405,764	$	119,919	$	285,845	$	—

- Definition

Tabular disclosure of financial instruments measured at fair value, including those classified in shareholders' equity measured on a recurring or nonrecurring basis. Disclosures include, but are not limited to, fair value measurements recorded and the reasons for the measurements, level within the fair value hierarchy in which the fair value measurements are categorized and transfers between levels 1 and 2. Nonrecurring fair value measurements are those that are required or permitted in the statement of financial position in particular circumstances.

+ References + Details

CASH EQUIVALENTS AND MARKETABLE SECURITIES - (Tables)

9 Months Ended

Sep. 30, 2022

Investments, Debt and Equity Securities [Abstract]

Marketable Securities

The table below summarizes the Company’s cash equivalents and marketable securities (in thousands):


	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses		Estimated Fair Value
September 30, 2022
Assets
Cash equivalents:
Money market funds	$	44,911	$	—	$	—	$	44,911


Total	44,911		—		—		44,911
Marketable securities:
U.S. government-sponsored entity debt securities	23,832		—		(440)		23,392
Commercial paper securities	120,750		11		(375)		120,386
Corporate debt securities	15,050		—		(162)		14,888
Asset-backed securities	38,039		2		(268)		37,773
U.S. treasury bills	16,943		—		(21)		16,922
Certificate of deposits	42,478		5		(146)		42,337
Total	257,092		18		(1,412)		255,698
Total cash equivalents and marketable securities	$	302,003	$	18	$	(1,412)	$	300,609

December 31, 2021
Assets
Cash equivalents:
Money market funds	$	119,919	$	—	$	—	$	119,919
Total	119,919		—		—		119,919
Marketable securities:
U.S. government-sponsored entity debt securities	30,700		1		(87)		30,614
Commercial paper securities	105,792		7		(42)		105,757
Corporate debt securities	33,723		1		(42)		33,682
Asset-backed securities	70,807		1		(107)		70,701
Certificate of deposits	45,116		1		(26)		45,091
Total	286,138		11		(304)		285,845
Total cash equivalents and marketable securities	$	406,057	$	11	$	(304)	$	405,764

Fair value of investments available-for-sale

The fair value of marketable securities by contractual maturity were as follows (in thousands):


	September 30, 2022		December 31, 2021
Maturing in one year or less	$	218,998	$	197,676
Maturing after one year through five years	36,700		88,169
Total	$	255,698	$	285,845

MAJOR CUSTOMERS, PARTNERSHIPS AND STRATEGIC ALLIANCES - (Tables)

9 Months Ended

Sep. 30, 2022

Novartis Institutes for BioMedical Research, Inc.

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Revenues Recognized under Agreement

Revenues recognized under the agreement were as follows (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2022		2021		2022		2021
Revenue related to Novartis agreement:
Recognition of upfront license fee	$	7,582	$	9,093	$	23,222	$	22,852
Research services	2,028		2,421		6,211		6,104
Total	$	9,610	$	11,514	$	29,433	$	28,956

Biogen MA, Inc.

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Revenues Recognized under Agreement

Revenues recognized under the agreement were as follows (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2022		2021		2022		2021
Revenue related to Biogen agreement:
Recognition of license and stand-ready fee	$	7,306	$	7,306	$	21,918	$	21,918
Research services	2,107		3,661		8,740		10,266
Total	$	9,413	$	10,967	$	30,658	$	32,184

Kite Pharma, Inc.

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Revenues Recognized under Agreement

Revenues recognized under the agreement were as follows (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2022		2021		2022		2021
Revenue related to Kite agreement:
Recognition of license and stand-ready fee	$	6,296	$	6,296	$	18,682	$	18,682
Research services	619		113		875		339
Total	$	6,915	$	6,409	$	19,557	$	19,021

Sanofi S.A.

Research and Development Arrangement, Contract to Perform for Others [Line Items]

Revenues Recognized under Agreement

Revenues recognized under the 2014 Collaboration Agreement were as follows (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2022		2021		2022		2021
Revenue related to Sanofi agreement:
Recognition of upfront fee	$	—	$	(1,125)	$	677	$	(692)
Research services	—		1,125		2,126		2,417
Milestone achievement	—		(759)		457		(467)
Total	$	—	$	(759)	$	3,260	$	1,258

STOCK-BASED COMPENSATION - (Tables)

9 Months Ended

Sep. 30, 2022

Share-Based Payment Arrangement [Abstract]

Stock-Based Compensation Expense

The following table shows total stock-based compensation expense included in the Condensed Consolidated Statements of Operations (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2022		2021		2022		2021
Research and development	$	4,395	$	4,935	$	13,656	$	14,645
General and administrative	3,398		2,938		9,746		10,235
Total stock-based compensation expense	$	7,793	$	7,873	$	23,402	$	24,880

ORGANIZATION, BASIS OF PRESENTATION, AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended		9 Months Ended		102 Months Ended
	Sep. 30, 2022	Sep. 30, 2021	Sep. 30, 2022	Sep. 30, 2021	Jun. 28, 2022
Change in Accounting Estimate [Line Items]
Decreased revenue	$ (26,460)	$ (28,563)	$ (84,069)	$ (82,715)
Increased net loss	$ 53,155	$ 47,688	$ 140,305	$ 140,801
Increased earnings per share, basic (in dollars per share)	$ (0.34)	$ (0.33)	$ (0.93)	$ (0.98)
Increased earnings per share, diluted (in dollars per share)	$ (0.34)	$ (0.33)	$ (0.93)	$ (0.98)
Letter of credit established as a deposit	$ 1,500		$ 1,500
Sanofi S.A.
Change in Accounting Estimate [Line Items]
Decreased revenue	$ 0	$ 759	$ (3,260)	$ (1,258)	$ (13,500)
Sanofi S.A. \| Change In Collaboration Agreement Scope \| Collaboration agreement
Change in Accounting Estimate [Line Items]
Decreased revenue		2,500		2,500
Increased net loss		$ 2,500		$ 2,500
Increased earnings per share, basic (in dollars per share)		$ 0.02		$ 0.02
Increased earnings per share, diluted (in dollars per share)		$ 0.02		$ 0.02

- Definition

The amount of net income (loss) for the period per each share of common stock or unit outstanding during the reporting period.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
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The amount of net income (loss) for the period available to each share of common stock or common unit outstanding during the reporting period and to each share or unit that would have been outstanding assuming the issuance of common shares or units for all dilutive potential common shares or units outstanding during the reporting period.

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The consolidated profit or loss for the period, net of income taxes, including the portion attributable to the noncontrolling interest.

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- Definition

Amount, excluding tax collected from customer, of revenue from satisfaction of performance obligation by transferring promised good or service to customer. Tax collected from customer is tax assessed by governmental authority that is both imposed on and concurrent with specific revenue-producing transaction, including, but not limited to, sales, use, value added and excise.

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ORGANIZATION, BASIS OF PRESENTATION, AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Revenues from Strategic Partnering Collaboration Agreements and Research Activity Grants (Detail) - Revenue from contract with customer - Customer concentration risk	3 Months Ended		9 Months Ended
	Sep. 30, 2022	Sep. 30, 2021	Sep. 30, 2022	Sep. 30, 2021
Novartis Institutes for BioMedical Research, Inc.
Concentration Risk [Line Items]
Percentage of revenues	36.00%	40.00%	35.00%	35.00%
Biogen MA, Inc.
Concentration Risk [Line Items]
Percentage of revenues	36.00%	38.00%	36.00%	39.00%
Kite Pharma, Inc.
Concentration Risk [Line Items]
Percentage of revenues	26.00%	22.00%	23.00%	23.00%
Sanofi S.A.
Concentration Risk [Line Items]
Percentage of revenues	0.00%	(3.00%)	4.00%	2.00%

ORGANIZATION, BASIS OF PRESENTATION, AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Reconciliation of Cash and Cash Equivalents (Detail) - USD ($) $ in Thousands	Sep. 30, 2022	Dec. 31, 2021	Sep. 30, 2021	Dec. 31, 2020
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Cash and cash equivalents	$ 94,570	$ 178,872	$ 176,954	$ 131,329
Non-current restricted cash	1,500	1,500	1,500	1,500
Cash, cash equivalents, and restricted cash as reported within the accompanying Condensed Consolidated Statements of Cash Flows	$ 96,070	$ 180,372	$ 178,454	$ 132,829

CASH EQUIVALENTS AND MARKETABLE SECURITIES - Summary of Cash Equivalents and Available-for-Sale Securities (Detail) - USD ($) $ in Thousands	Sep. 30, 2022	Dec. 31, 2021	Sep. 30, 2021	Dec. 31, 2020
ASSETS
Cash equivalents, Amortized Cost	$ 94,570	$ 178,872	$ 176,954	$ 131,329
Available-for-sale securities, Amortized Cost	257,092	286,138
Available-for-sale securities, Gross Unrealized Gains	18	11
Available-for-sale securities, Gross Unrealized (Losses)	(1,412)	(304)
Available-for-sale securities, Estimated Fair Value	255,698	285,845
Total cash equivalents and available-for-sale securities, Amortized Cost	302,003	406,057
Total cash equivalents and available-for-sale securities, Gross Unrealized Gains	18	11
Total cash equivalents and available-for-sale securities, Gross Unrealized (Losses)	(1,412)	(304)
Total cash equivalents and available-for-sale securities, Estimated Fair Value	300,609	405,764
Money market funds
ASSETS
Cash equivalents, Amortized Cost	44,911	119,919
Cash equivalents, Gross Unrealized Gains	0	0
Cash equivalents, Gross Unrealized (Losses)	0	0
Cash equivalents, Estimated Fair Value	44,911	119,919
Cash equivalents
ASSETS
Cash equivalents, Amortized Cost	44,911	119,919
Cash equivalents, Gross Unrealized Gains	0	0
Cash equivalents, Gross Unrealized (Losses)	0	0
Cash equivalents, Estimated Fair Value	44,911	119,919
U.S. government-sponsored entity debt securities
ASSETS
Available-for-sale securities, Amortized Cost	23,832	30,700
Available-for-sale securities, Gross Unrealized Gains	0	1
Available-for-sale securities, Gross Unrealized (Losses)	(440)	(87)
Available-for-sale securities, Estimated Fair Value	23,392	30,614
Commercial paper securities
ASSETS
Available-for-sale securities, Amortized Cost	120,750	105,792
Available-for-sale securities, Gross Unrealized Gains	11	7
Available-for-sale securities, Gross Unrealized (Losses)	(375)	(42)
Available-for-sale securities, Estimated Fair Value	120,386	105,757
Corporate debt securities
ASSETS
Available-for-sale securities, Amortized Cost	15,050	33,723
Available-for-sale securities, Gross Unrealized Gains	0	1
Available-for-sale securities, Gross Unrealized (Losses)	(162)	(42)
Available-for-sale securities, Estimated Fair Value	14,888	33,682
Asset-backed securities
ASSETS
Available-for-sale securities, Amortized Cost	38,039	70,807
Available-for-sale securities, Gross Unrealized Gains	2	1
Available-for-sale securities, Gross Unrealized (Losses)	(268)	(107)
Available-for-sale securities, Estimated Fair Value	37,773	70,701
U.S. treasury bills
ASSETS
Available-for-sale securities, Amortized Cost	16,943
Available-for-sale securities, Gross Unrealized Gains	0
Available-for-sale securities, Gross Unrealized (Losses)	(21)
Available-for-sale securities, Estimated Fair Value	16,922
Certificates of deposit
ASSETS
Available-for-sale securities, Amortized Cost	42,478	45,116
Available-for-sale securities, Gross Unrealized Gains	5	1
Available-for-sale securities, Gross Unrealized (Losses)	(146)	(26)
Available-for-sale securities, Estimated Fair Value	$ 42,337	$ 45,091

CASH EQUIVALENTS AND MARKETABLE SECURITIES - Summary of cost and estimated fair value of short-term investments (Details) - USD ($) $ in Thousands	Sep. 30, 2022	Dec. 31, 2021
Investments, Debt and Equity Securities [Abstract]
Maturing in one year or less	$ 218,998	$ 197,676
Maturing after one year through five years	36,700	88,169
Total	$ 255,698	$ 285,845

CASH EQUIVALENTS AND MARKETABLE SECURITIES - Additional Information (Details) - USD ($)	3 Months Ended		9 Months Ended
	Sep. 30, 2022	Sep. 30, 2021	Sep. 30, 2022	Sep. 30, 2021	Dec. 31, 2021
Investments, Debt and Equity Securities [Abstract]
Realized investment gains (losses)	$ 0	$ 0	$ 0	$ 0
Allowance for credit loss related to marketable securities, not previously recorded	0	0	0	0
Other impairment charges related to marketable securities	0	$ 0	0	$ 0
Allowance for credit loss related to marketable securities	$ 0		$ 0		$ 0

BASIC AND DILUTED NET LOSS PER SHARE - Additional Information (Detail) - shares	9 Months Ended
	Sep. 30, 2022	Sep. 30, 2021
Earnings Per Share [Abstract]
Stock options and RSUs outstanding (in shares)	18,524,016	15,838,002

MAJOR CUSTOMERS, PARTNERSHIPS AND STRATEGIC ALLIANCES - Novartis Institutes for BioMedical Research, Inc. (Detail) - USD ($) $ in Thousands		1 Months Ended	3 Months Ended		6 Months Ended	9 Months Ended		12 Months Ended
	Jul. 27, 2020	Aug. 31, 2020	Sep. 30, 2022	Sep. 30, 2021	Jun. 30, 2021	Sep. 30, 2022	Sep. 30, 2021	Dec. 31, 2020	Dec. 31, 2021
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Accounts receivable			$ 4,522			$ 4,522			$ 6,013
Novartis Institutes for BioMedical Research, Inc.
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Contract asset		$ 1,500
Novartis Institutes for BioMedical Research, Inc. \| Collaboration And License Agreement
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Proceeds from collaborators		$ 75,000
Collaborative arrangement transaction price	$ 95,100
License fee	75,000
Collaborative arrangement estimated reimbursable service costs	20,100
Accounts receivable			2,000			2,000			1,900
Deferred revenue			17,700			17,700			$ 40,900
Financial advisory fees					$ 1,500
Percent of initial recognition								2.00%
Amortization			$ 200	$ 200		$ 500	$ 500
Novartis Institutes for BioMedical Research, Inc. \| Collaboration And License Agreement \| Achievement Of Specified Preclinical Development Clinical Development And First Commercial Sale Milestones
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Development and sales-based milestone payments to be received	420,000
Novartis Institutes for BioMedical Research, Inc. \| Collaboration And License Agreement \| Achievement Of Commercial Milestones
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Development and sales-based milestone payments to be received	$ 300,000

MAJOR CUSTOMERS, PARTNERSHIPS AND STRATEGIC ALLIANCES - Revenues Recognized under Agreement (Detail) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended		102 Months Ended
	Sep. 30, 2022	Sep. 30, 2021	Sep. 30, 2022	Sep. 30, 2021	Jun. 28, 2022
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues	$ 26,460	$ 28,563	$ 84,069	$ 82,715
Novartis Institutes for BioMedical Research, Inc.
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues	9,610	11,514	29,433	28,956
Novartis Institutes for BioMedical Research, Inc. \| Recognition of upfront fee
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues	7,582	9,093	23,222	22,852
Novartis Institutes for BioMedical Research, Inc. \| Research services
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues	2,028	2,421	6,211	6,104
Biogen MA, Inc.
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues	9,413	10,967	30,658	32,184
Biogen MA, Inc. \| Recognition of upfront fee
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues	7,306	7,306	21,918	21,918
Biogen MA, Inc. \| Research services
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues	2,107	3,661	8,740	10,266
Kite Pharma, Inc.
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues	6,915	6,409	19,557	19,021
Kite Pharma, Inc. \| Recognition of upfront fee
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues	6,296	6,296	18,682	18,682
Kite Pharma, Inc. \| Research services
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues	619	113	875	339
Sanofi
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues	0	(759)	3,260	1,258	$ 13,500
Sanofi \| Recognition of upfront fee
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues	0	(1,125)	677	(692)
Sanofi \| Research services
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues	0	1,125	2,126	2,417
Sanofi \| Milestone achievement
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues	$ 0	$ (759)	$ 457	$ (467)

- Definition

+ References

+ Details

MAJOR CUSTOMERS, PARTNERSHIPS AND STRATEGIC ALLIANCES - Biogen MA, Inc. (Detail) $ / shares in Units, $ in Thousands	1 Months Ended		3 Months Ended		9 Months Ended		12 Months Ended
	May 31, 2020 USD ($)	Apr. 30, 2020 USD ($) product_target $ / shares shares	Sep. 30, 2022 USD ($)	Sep. 30, 2021 USD ($)	Sep. 30, 2022 USD ($)	Sep. 30, 2021 USD ($)	Dec. 31, 2020 USD ($)	Dec. 31, 2021 USD ($)
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Accounts receivable			$ 4,522		$ 4,522			$ 6,013
Biogen MA, Inc. \| Stock purchase agreement
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Number of shares issued in transaction (in shares) \| shares		24,420,157
Sale of stock, price per share (in dollars per share) \| $ / shares		$ 9.2137
Consideration received on transaction		$ 225,000
Standstill restriction period		3 years
Standstill restriction, ownership threshold percentage ownership percentage		5.00%
Collaborative arrangement transaction price		$ 204,600
Excess consideration received on transaction		79,600
Collaboration agreement, equity issued		145,400
Accounts receivable			1,700		1,700			2,800
Deferred revenue			132,100		132,100			$ 154,000
Recognized portion of equity issued							$ 2,900
Biogen MA, Inc. \| Collaboration And License Agreement
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Consideration received on transaction		204,600
Proceeds from collaborators	$ 125,000	125,000
Maximum milestone payment receivable		$ 2,400,000
Number of additional product targets \| product_target		7
Remaining period		5 years
Number of product targets selected \| product_target		3
Research period		7 years
Maximum number of product targets replaced \| product_target		10
Financial advisory fees							$ 7,000
Percent of initial recognition		2.00%
Portion of contract asset recognized			4,100		4,100
Amortization			$ 100	$ 100	$ 400	$ 400
Biogen MA, Inc. \| Collaboration And License Agreement \| Pre-approval milestone
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Maximum milestone payment receivable		$ 925,000
Biogen MA, Inc. \| Collaboration And License Agreement \| Sales-based milestone
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Maximum milestone payment receivable		$ 1,500,000

MAJOR CUSTOMERS, PARTNERSHIPS AND STRATEGIC ALLIANCES - Kite Pharma, Inc. (Detail) $ in Thousands	1 Months Ended
	Sep. 30, 2019 USD ($)	Apr. 30, 2018 USD ($) milestone option	Sep. 30, 2022 USD ($)	Dec. 31, 2021 USD ($)
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Accounts receivable			$ 4,522	$ 6,013
Kite Pharma, Inc. \| Collaboration And License Agreement
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Milestone payments received		$ 150,000
Development and sales-based milestone payments to be received		$ 3,000,000
Initial research term of agreement		6 years
Number of options to extend initial research term \| option		2
Extended research term of agreement		1 year
Separate upfront fee		$ 10,000
Collaborative arrangement estimated reimbursable service costs for new research plan	$ 3,400
Collaborative arrangement transaction price	189,300
Revenues under agreement	150,000
Collaborative arrangement estimated reimbursable service costs	$ 39,300
Accounts receivable			600	100
Deferred revenue			$ 37,800	$ 56,500
Kite Pharma, Inc. \| Collaboration And License Agreement \| Achievement of specified research, clinical development, regulatory and first commercial sale milestones
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Contingent development - and sales-based milestone payments to be received		1,300,000
Kite Pharma, Inc. \| Collaboration And License Agreement \| Achievement of specified sales-based milestones if annual worldwide net sales of licensed products reach specified levels
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Contingent development - and sales-based milestone payments to be received		$ 1,800,000
Maximum amount of achieved milestones to receive payment \| milestone		10

MAJOR CUSTOMERS, PARTNERSHIPS AND STRATEGIC ALLIANCES - Sanofi S.A. (Detail) $ / shares in Units, $ in Thousands		1 Months Ended	3 Months Ended		9 Months Ended		102 Months Ended
	Sep. 06, 2022 USD ($)	Jan. 31, 2014 USD ($) program	Sep. 30, 2022 USD ($) $ / shares	Sep. 30, 2021 USD ($) $ / shares	Sep. 30, 2022 USD ($) milestone product $ / shares	Sep. 30, 2021 USD ($) $ / shares	Jun. 28, 2022 USD ($)	Dec. 31, 2021 USD ($)
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues			$ 26,460	$ 28,563	$ 84,069	$ 82,715
Accounts receivable			4,522		4,522			$ 6,013
Decrease in research and development expense			900		900
Increased net loss			$ 53,155	$ 47,688	$ 140,305	$ 140,801
Increased earnings per share, diluted (in dollars per share) \| $ / shares			$ (0.34)	$ (0.33)	$ (0.93)	$ (0.98)
Increased earnings per share, basic (in dollars per share) \| $ / shares			$ (0.34)	$ (0.33)	$ (0.93)	$ (0.98)
Sanofi S.A.
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Number of research programs \| program		2
Revenues under agreement		$ 20,000
Revenues			$ 0	$ (759)	$ 3,260	$ 1,258	$ 13,500
Number of products approved \| product					0
Post transition assistance period	90 days
Expenses reimbursable through December 31, 2023 (up to)	$ 7,000
Expenses reimbursable after December 31, 2023 (up to)	$ 5,300
Sanofi S.A. \| Collaboration And License Agreement
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues under agreement							20,000
Number of milestones included in transaction price \| milestone					0
Collaborative arrangement transaction price							96,300
Collaborative arrangement estimated reimbursable service costs							62,800
Accounts receivable			0		$ 0			600
Deferred revenue			$ 0		$ 0			$ 1,100
Sanofi S.A. \| Collaboration And License Agreement \| Milestone three
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Milestone revenue receivable							$ 13,500
Sanofi S.A. \| Collaboration agreement \| Change In Collaboration Agreement Scope
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues				(2,500)		(2,500)
Increased net loss				$ 2,500		$ 2,500
Increased earnings per share, diluted (in dollars per share) \| $ / shares				$ 0.02		$ 0.02
Increased earnings per share, basic (in dollars per share) \| $ / shares				$ 0.02		$ 0.02

MAJOR CUSTOMERS, PARTNERSHIPS AND STRATEGIC ALLIANCES - Pfizer Inc. (Detail)	1 Months Ended			3 Months Ended		9 Months Ended		12 Months Ended	52 Months Ended	65 Months Ended
	Sep. 30, 2020 USD ($)	Dec. 31, 2017 USD ($)	May 31, 2017 USD ($)	Sep. 30, 2022 USD ($)	Sep. 30, 2021 USD ($)	Sep. 30, 2022 USD ($)	Sep. 30, 2021 USD ($)	Dec. 31, 2020 USD ($)	Mar. 31, 2022 USD ($) milestone product	Sep. 30, 2022 USD ($) milestone product
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues				$ 26,460,000	$ 28,563,000	$ 84,069,000	$ 82,715,000
Pfizer Inc.
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Agreement termination, term			15 years
Pfizer Inc. \| S B Five Two Five And Other Products
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues under agreement			$ 70,000,000
Potential amount to be funded for achievement of specified commercialized and sales milestones			266,500,000
Milestone revenue receivable			300,000,000
Pfizer Inc. \| S B Five Two Five And Other Products \| Achievement of specified clinical development intellectual property and regulatory milestones
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Development and sales-based milestone payments to be received			208,500,000
Pfizer Inc. \| S B Five Two Five And Other Products \| Achievement of first commercial sale milestones
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Development and sales-based milestone payments to be received			475,000,000
Pfizer Inc. \| Other products
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Milestone revenue receivable			175,000,000
Pfizer Inc. \| C9ORF72
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues under agreement		$ 5,000,000						$ 5,000,000
Development and sales-based milestone payments to be received	$ 5,000,000
Collaborative arrangement transaction price		$ 17,000,000
Pfizer SB-525
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues under agreement										$ 55,000,000
Collaborative arrangement transaction price			134,000,000
Research service fees			$ 79,000,000
Revenues				0	0	0	0
Pfizer SB-525 \| S B Five Two Five And Other Products
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Collaborative arrangement, number of milestones achieved \| milestone										2
Milestone payments received										$ 55,000,000
Number of products approved \| product										0
Number of milestones included in transaction price \| milestone									0
Pfizer C9ORF72
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Agreement termination, term		15 years
Milestone payments received		$ 12,000,000							$ 5,000,000
Number of products approved \| product									0
Number of milestones included in transaction price \| milestone									0
Pfizer C9ORF72 \| C9ORF72
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Revenues under agreement		12,000,000
Revenues				$ 0	$ 0	$ 0	$ 0
Pfizer C9ORF72 \| C9ORF72 \| Achievement Of Specified Preclinical Development Clinical Development And First Commercial Sale Milestones \| Maximum
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Development and sales-based milestone payments to be received		60,000,000
Pfizer C9ORF72 \| C9ORF72 \| Achievement Of Commercial Milestones \| Maximum
Research and Development Arrangement, Contract to Perform for Others [Line Items]
Development and sales-based milestone payments to be received		$ 90,000,000

STOCK-BASED COMPENSATION - Stock-Based Compensation Expense (Detail) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2022	Sep. 30, 2021	Sep. 30, 2022	Sep. 30, 2021
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Total stock-based compensation expense	$ 7,793	$ 7,873	$ 23,402	$ 24,880
Research and development
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Total stock-based compensation expense	4,395	4,935	13,656	14,645
General and administrative
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Total stock-based compensation expense	$ 3,398	$ 2,938	$ 9,746	$ 10,235

STOCKHOLDERS' EQUITY (Detail) - Jefferies LLC - At-The-Market Offering Agreement - USD ($) $ in Millions	1 Months Ended		3 Months Ended		9 Months Ended
	Oct. 31, 2022	Aug. 31, 2020	Sep. 30, 2022	Sep. 30, 2021	Sep. 30, 2022	Sep. 30, 2021
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Stock offering program, maximum value		$ 150.0
Issuance of common stock under public offering, net of issuance costs (in shares)			8,483,104	202,705	14,711,770	2,007,932
Consideration received on transaction			$ 42.2	$ 2.4	$ 66.4	$ 27.1
Subsequent Event
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Consideration received on transaction	$ 0.6

INCOME TAXES (Details) - USD ($)	3 Months Ended		9 Months Ended
INCOME TAXES (Details) - USD ($)	Sep. 30, 2022	Sep. 30, 2021	Sep. 30, 2022	Sep. 30, 2021
Income Tax Disclosure [Abstract]
Income tax expense	$ 30,000	$ 86,000	$ 170,000	$ 373,000
Employee retention credit, CARES Act	0		3,000,000
Proceeds from employee retention credit, CARES Act	$ 0		$ 0

- Definition

Amount of current income tax expense (benefit) and deferred income tax expense (benefit) pertaining to continuing operations.

+ References + Details

FAIR VALUE MEASUREMENTS (Detail) - USD ($) $ in Thousands	Sep. 30, 2022	Dec. 31, 2021
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	$ 255,698	$ 285,845
Money market funds
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total cash equivalents	44,911	119,919
U.S. government-sponsored entity debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	23,392	30,614
Commercial paper securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	120,386	105,757
Corporate debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	14,888	33,682
Asset-backed securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	37,773	70,701
U.S. treasury bills
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	16,922
Certificates of deposit
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	42,337	45,091
Fair value on recurring basis
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total cash equivalents	44,911	119,919
Total marketable securities	255,698	285,845
Total cash equivalents and marketable securities and free shares asset	300,609	405,764
Fair value on recurring basis \| Money market funds
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total cash equivalents	44,911	119,919
Fair value on recurring basis \| U.S. government-sponsored entity debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	23,392	30,614
Fair value on recurring basis \| Commercial paper securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	120,386	105,757
Fair value on recurring basis \| Corporate debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	14,888	33,682
Fair value on recurring basis \| Asset-backed securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	37,773	70,701
Fair value on recurring basis \| U.S. treasury bills
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	16,922
Fair value on recurring basis \| Certificates of deposit
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	42,337	45,091
Fair value on recurring basis \| Level 1
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total cash equivalents	44,911	119,919
Total marketable securities	0	0
Total cash equivalents and marketable securities and free shares asset	44,911	119,919
Fair value on recurring basis \| Level 1 \| Money market funds
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total cash equivalents	44,911	119,919
Fair value on recurring basis \| Level 1 \| U.S. government-sponsored entity debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	0	0
Fair value on recurring basis \| Level 1 \| Commercial paper securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	0	0
Fair value on recurring basis \| Level 1 \| Corporate debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	0	0
Fair value on recurring basis \| Level 1 \| Asset-backed securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	0	0
Fair value on recurring basis \| Level 1 \| U.S. treasury bills
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	0
Fair value on recurring basis \| Level 1 \| Certificates of deposit
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	0	0
Fair value on recurring basis \| Level 2
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total cash equivalents	0	0
Total marketable securities	255,698	285,845
Total cash equivalents and marketable securities and free shares asset	255,698	285,845
Fair value on recurring basis \| Level 2 \| Money market funds
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total cash equivalents	0	0
Fair value on recurring basis \| Level 2 \| U.S. government-sponsored entity debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	23,392	30,614
Fair value on recurring basis \| Level 2 \| Commercial paper securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	120,386	105,757
Fair value on recurring basis \| Level 2 \| Corporate debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	14,888	33,682
Fair value on recurring basis \| Level 2 \| Asset-backed securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	37,773	70,701
Fair value on recurring basis \| Level 2 \| U.S. treasury bills
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	16,922
Fair value on recurring basis \| Level 2 \| Certificates of deposit
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	42,337	45,091
Fair value on recurring basis \| Level 3
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total cash equivalents	0	0
Total marketable securities	0	0
Total cash equivalents and marketable securities and free shares asset	0	0
Fair value on recurring basis \| Level 3 \| Money market funds
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total cash equivalents	0	0
Fair value on recurring basis \| Level 3 \| U.S. government-sponsored entity debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	0	0
Fair value on recurring basis \| Level 3 \| Commercial paper securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	0	0
Fair value on recurring basis \| Level 3 \| Corporate debt securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	0	0
Fair value on recurring basis \| Level 3 \| Asset-backed securities
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	0	0
Fair value on recurring basis \| Level 3 \| U.S. treasury bills
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	0
Fair value on recurring basis \| Level 3 \| Certificates of deposit
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Total marketable securities	$ 0	$ 0