Sangamo Highlights Advancements in Genomic Medicine Pipeline and Expanded R&D and Manufacturing Capabilities at R&D Day
- IND transfer to Pfizer for SB-525 hemophilia A gene therapy is substantially completed; Pfizer is advancing SB-525 into a Phase 3 registrational study in 2020
- At R&D Day, Sangamo is detailing global capabilities across clinical science, operations, product development, and manufacturing
- Company is also introducing new gene therapy and genome regulation programs for clinical development, including several addressing highly prevalent diseases, with IND targets in 2021 and 2022
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“The talent, R&D capabilities, manufacturing expertise, and operations infrastructure we have brought to Sangamo have enabled us to advance a genomic medicine pipeline that spans multiple therapeutic areas and now also extends into late-stage development,” said
Macrae continued: “We will continue to pursue a dual approach of retaining certain programs for our proprietary pipeline while also establishing pharmaceutical partnerships to gain access to therapeutic area expertise and financial, operational, and commercial resources. Strategic collaborations will be a particularly important consideration as we advance programs for diseases affecting large patient populations.”
R&D Day updates on clinical and preclinical pipeline programs:
Gene therapy product candidates for hemophilia A, Fabry disease, and PKU
SB-525 is a gene therapy product candidate for hemophilia A being developed by Sangamo and Pfizer under a global development and commercialization collaboration agreement. The transfer of the SB-525 IND to Pfizer is substantially completed. Pfizer is advancing SB-525 into a Phase 3 registrational study in 2020 and has recently begun enrolling patients into a Phase 3 lead-in study.
At R&D Day, Sangamo executives are presenting data from the SB-525 program which were recently announced at the
The cassette engineering, AAV engineering and manufacturing expertise which Sangamo used in the development of SB-525 are also being applied to the ST-920 Fabry disease program, which is being evaluated in a Phase 1/2 clinical trial, as well as to the newly announced ST-101 gene therapy program for PKU, which is being evaluated in preclinical studies with a planned IND submission in 2021.
Engineered ex vivo cell therapy candidates for beta thalassemia, kidney transplantation, and preclinical research in multiple sclerosis (MS)
Sangamo is providing an overview of the Company’s diversified cell therapy pipeline this morning. Cell therapy incorporates Sangamo’s experience and core strengths, including cell culture and engineering, gene editing, and AAV manufacturing. At R&D Day, Sangamo scientists today are reviewing the early data presented this month at ASH from the ST-400 beta thalassemia ex vivo gene-edited cell therapy program, which is being developed in partnership with
Sangamo is also providing updates on the company’s CAR-TREG clinical and preclinical programs. CAR-TREGS are regulatory T cells equipped with a chimeric antigen receptor. Sangamo is the pioneer in CAR-TREGS, which may have the potential to treat inflammatory and autoimmune diseases. TX200 is being evaluated in the STEADFAST study, the first ever clinical trial evaluating a CAR-TREG cell therapy. Tx200 is being developed for the prevention of immune-mediated organ rejection in patients who have received a kidney transplant, a significant unmet medical need. Results from this trial will provide data on safety and proof of mechanism, building a critical understanding of CAR-TREGS in patients, and may provide a gateway to autoimmune indications such as Crohn’s disease and multiple sclerosis (MS). Sangamo is also presenting preclinical murine data demonstrating that CAR-TREGS accumulate and proliferate in the CNS and reduce a marker of MS.
In vivo genome editing optimization
Clinical data presented earlier this year provided evidence that Sangamo had successfully edited the genome of patients with mucopolysaccharidosis type II (MPS II) but also suggested that the zinc finger nuclease in vivo gene editing reagents were under-dosed using first-generation technology. Sangamo has identified potential improvements that may enhance the potency of in vivo genome editing, including increasing total AAV vector dose, co-packaging both ZFNs in one AAV vector, and engineering second-generation AAVs, ZFNs, and donor transgenes.
Genome regulation pipeline candidates targeting neurodegenerative diseases including Alzheimer’s and Parkinson’s
Sangamo scientists today are presenting data demonstrating that the company’s engineered zinc finger protein transcription factors (ZFP-TFs) specifically and powerfully repress key genes involved in brain diseases including Alzheimer’s, Parkinson’s, Huntington’s, ALS, and Prion diseases. Sangamo is advancing its first two genome regulation programs toward clinical development:
- ST-501 for tauopathies including Alzheimer’s, with an IND anticipated in 2021
- ST-502 for alpha-synuclein diseases including Parkinson’s, with an IND anticipated in 2022
Sangamo scientists are also presenting data demonstrating progress in the development of new AAV serotypes for use in CNS diseases.
Manufacturing capabilities and strategy
Sangamo is nearing completion of its buildout of a GMP manufacturing facility at the new Company headquarters in
R&D Day webcast
A live webcast of the R&D Day, including audio and slides, will be available on the Events and Presentations page of the Sangamo website today at
Sangamo Forward Looking Statements
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