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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, DC 20549
FORM 8-K
CURRENT REPORT PURSUANT
TO SECTION 13 OR 15(D) OF THE
SECURITIES EXCHANGE ACT OF 1934
Date of report (Date of earliest event reported): April 4, 2005
SANGAMO BIOSCIENCES, INC.
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(Exact Name of Registrant as Specified in Its Charter)
Delaware
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(State or Other Jurisdiction of Incorporation)
000-30171 68-0359556
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(Commission File Number) (IRS Employer Identification No.)
501 Canal Blvd, Suite A100 Richmond, California 94804
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(Address of Principal Executive Offices) (Zip Code)
(510) 970-6000
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(Registrant's Telephone Number, Including Area Code)
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(Former Name or Former Address, if Changed Since Last Report)
Check the appropriate box below if the Form 8-K filing is intended to
simultaneously satisfy the filing obligation of the registrant under any of the
following provisions (see General Instruction A.2. below):
[ ] Written communications pursuant to Rule 425 under the Securities
Act (17 CFR 230.425)
[ ] Soliciting material pursuant to Rule 14a-12 under the Exchange
Act (17 CFR 240.14a-12)
[ ] Pre-commencement communications pursuant to Rule 14d-2(b) under the
Exchange Act (17 CFR 240.14d-2(b))
[ ] Pre-commencement communications pursuant to Rule 13e-4(c) under the
Exchange Act (17 CFR 240.13e-4(c))
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ITEM 8.01 OTHER EVENTS
On April 4, 2005, Sangamo BioSciences Inc. issued a press release
announcing that data from their gene correction program was published in the
online edition of the scientific journal Nature.
A copy of the press release issued by Sangamo BioSciences, Inc. relating to this
event is filed as an exhibit to this Current Report on Form 8-K.
ITEM 9.01 FINANCIAL STATEMENTS AND EXHIBITS
(c) Exhibits. The following material is filed as an exhibit to this Current
Report on Form 8-K:
Exhibit No.
99.1 Press Release Issued April 4, 2005.
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the
Registrant has duly caused this report to be signed on its behalf by the
undersigned hereunto duly authorized.
DATE: April 4, 2005
SANGAMO BIOSCIENCES, INC.
By: /s/ EDWARD O. LANPHIER II
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Edward O. Lanphier II
President, Chief Executive Officer
Exhibit 99.1
SANGAMO BIOSCIENCES ANNOUNCES PUBLICATION IN NATURE DEMONSTRATING THE USE
OF ITS ZINC FINGER TECHNOLOGY TO CORRECT HUMAN GENES
PAPER DESCRIBES APPROACH FOR 'GENOME EDITING' THAT MAY LEAD TO POTENTIAL
TREATMENT FOR MONOGENIC DISEASES AND HIV/AIDS
RICHMOND, Calif., April 4 /PRNewswire-FirstCall/ -- Sangamo BioSciences,
Inc. (Nasdaq: SGMO) today announced publication of data that demonstrates the
use of the Company's zinc finger DNA-binding protein (ZFP) technology to achieve
highly efficient, permanent correction of a disease-causing gene in primary
human cells. This research, published in Nature as an advance online
publication, represents a significant advance in the ability to specifically and
efficiently modify the human genome and provides the scientific foundation for
potential therapeutic approaches for a variety of genetic disorders and
infectious diseases.
In this study, Sangamo scientists demonstrated the use of engineered zinc
finger nucleases (ZFN(TM)) to correct errors in the DNA sequence of a
disease-causing gene, the IL2Rgamma gene. Correction was achieved in a high
percentage of treated cells without the need for selection. Importantly, gene
correction was permanent and eliminated the need for integration of any foreign
DNA sequence, a cause of problems in certain gene therapy studies.
"Using our ZFN technology we have advanced the field of targeted homologous
recombination to levels of efficiency and specificity that could make potential
therapeutic applications feasible," stated Michael C. Holmes, Ph.D., Director,
Therapeutic Gene Modification at Sangamo and the study's senior author. "Our ZFN
technology allows us to facilitate modification of a DNA sequence at a very
specific point in the genome, in this case, at the site of a mutation in a gene.
The cell's own machinery corrects the mutation using a DNA sequence that we
provide. All of this happens without the need for integration of foreign DNA
into the genome of cells. Once the gene is repaired, these cells undergo normal
division and replication, resulting in daughter cells that carry the modified
gene and thus are permanently corrected."
"For years, scientists have been searching for a way to modify or edit the
genome of plants and animals in a precise and predictable fashion," said Nobel
Laureate, Professor Sir Aaron Klug, of the MRC Laboratory of Molecular Biology,
Cambridge, UK. "This work is therefore truly a landmark study that provides the
foundation for gene modification-based therapeutics without the safety issues
that have plagued many traditional gene therapy applications. It gives me great
personal satisfaction to see this remarkable outcome of my original discovery of
zinc fingers and their development."
Study Results
Mutations in the gene encoding the IL2Rgamma protein invariably cause
X-linked SCID (X-linked Severe Combined Immunodeficiency Disease) or so-called
Bubble-boy disease. Patients with such mutations do not produce a functional
IL2Rgamma protein; never develop a functional immune system and die of severe
infections within 12-18 months of birth.
In this study, highly specific engineered ZFNs designed to bind to
sequences close to an X-linked SCID-causing mutation in the IL2Rgamma gene
resulted in a high percentage of the cells undergoing gene correction. In
addition, it was observed that approximately one third of the corrected cells
acquired the desired modification on both chromosomes. The expected downstream
changes in both RNA and protein levels were also observed. Comparably high
levels of correction were observed in primary human T-cells. While further work
will be required to optimize the system for therapeutic use, the gene correction
efficiencies established here may be sufficient to achieve a therapeutic effect.
"I would like to congratulate all of the Sangamo scientists involved in the
generation of these data," stated Edward Lanphier, Sangamo's president and CEO.
"It is gratifying that their work has been recognized by the prestigious journal
Nature. These results highlight the potential for gene correction therapy for
human monogenic disorders i.e. those diseases caused by mutation of a single
gene. We are now working with our clinical collaborators to move this technology
into the clinic. Our initial research will focus on monogenic diseases of blood
cells such as Sickle Cell Anemia and beta-Thalassemia. In addition, the
technology also forms the basis of our program to develop a potential
therapeutic for HIV infection by disrupting expression of the CCR5 gene to
generate a population of HIV-resistant cells."
About Zinc Finger DNA Binding Proteins
Zinc Finger DNA-binding Proteins (ZFPs) are a naturally occurring class of
DNA binding proteins. The DNA recognition and binding function of ZFPs can be
engineered and thus directed to a targeted sequence of DNA. This permits the
delivery of a variety of functional domains to a gene-specific location. ZFPs
are being developed for two significant therapeutic applications: gene
regulation and gene modification. In the case of therapeutic gene regulation,
ZFPs are being engineered to either turn on therapeutically beneficial genes or
turn off the expression of disease-causing genes. For gene modification, ZFPs
are being used in combination with a DNA cutting enzyme (endonuclease)
functional domain to generate ZFNs that facilitate the correction of mutant gene
sequences that cause disease or the disruption of genes that facilitate disease
progression.
About Sangamo
Sangamo BioSciences, Inc. is focused on the research and development of
novel DNA-binding proteins for therapeutic gene regulation and modification. The
most advanced ZFP Therapeutic(TM) development programs are currently in Phase I
clinical trials for evaluation of safety in patients with peripheral artery
disease and diabetic neuropathy. Other therapeutic development programs are
focused on ischemic heart disease, congestive heart failure, cancer, neuropathic
pain, and infectious and monogenic diseases. Sangamo's core competencies enable
the engineering of a class of DNA-binding proteins known as zinc finger
DNA-binding proteins (ZFPs). By engineering ZFPs that recognize
a specific DNA sequence Sangamo has created ZFP transcription factors (ZFP
TF(TM)) that can control gene expression and, consequently, cell function.
Sangamo is also developing sequence-specific ZFP Nucleases (ZFNs) for
therapeutic gene modification as a treatment and possible cure for a variety of
monogenic diseases such as sickle cell anemia and for infectious diseases such
as HIV. For more information about Sangamo, visit the company's web site at
www.sangamo.com or www.expressinglife.com.
This press release may contain forward-looking statements based on
Sangamo's current expectations. These forward-looking statements include,
without limitation, references to the research and development of novel ZFP TFs
and ZFNs, clinical trials and therapeutic applications of Sangamo's ZFP
technology platform. Actual results may differ materially from these
forward-looking statements due to a number of factors, including technological
challenges, Sangamo's ability to develop commercially viable products and
technological developments by our competitors. See the company's SEC filings,
and in particular, the risk factors described in the company's Annual Report on
Form 10-K and its most recent 10-Q. Sangamo assumes no obligation to update the
forward-looking information contained in this press release.
SOURCE Sangamo BioSciences, Inc.
-0- 04/04/2005
/CONTACT: Elizabeth Wolffe, Ph.D., of Sangamo BioSciences, Inc.,
+1-510-970-6000, ext. 271, or ewolffe@sangamo.com; or media, Kathy Nugent,
+1-212-213-0006, or investors, John Cummings, +1-415-352-6262, both of Burns
McClellan, Inc., for Sangamo BioSciences, Inc./
/Web site: http://www.sangamo.com