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
- Live webcast today at 8am Eastern Time
December 17, 2019 06:45 AM Eastern Standard Time
BRISBANE, Calif.--(BUSINESS WIRE)--Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, is hosting an R&D Day today beginning at 8am Eastern Time. During the event, Sangamo executives and scientists plan to provide updates across the Company?s clinical and preclinical pipeline, as well as an overview of manufacturing capabilities to support clinical and commercial supply. A live webcast link will be available on the?Events and Presentations?page of the Sangamo website
?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 Sandy Macrae, CEO of Sangamo. ?As we make progress in clinical development, we gain insights into the use of our technology and are applying those insights as we advance new programs, such as the gene therapy for PKU and the genome regulation candidates for CNS diseases we are announcing today.? 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 American Society of Hematology (ASH) annual meeting. 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 Sanofi. 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:?As we make progress in clinical development, we gain insights into the use of our technology and are applying those insights as we advance new programs, such as the gene therapy for PKU and the genome regulation candidates for CNS diseases we are announcing today.?
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- 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