Sangamo Therapeutics, Inc. Announces Upcoming Clinical Data Presentations At WORLDSymposium 2019
January 04, 2019 at 08:00 am EST
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Sangamo Therapeutics, Inc. announced that interim clinical data from the company's inherited metabolic diseases development programs will be presented at WORLDSymposium, an annual conference dedicated to lysosomal diseases being held February 4-8, 2019 at the Hyatt Regency Orlando in Orlando, Florida. The WORLDSymposium program includes two platform presentations from clinical trials of Sangamo's zinc finger nuclease (ZFN)-mediated in vivo genome editing product candidates, SB-318 and SB-913, which are being evaluated for the treatment of mucopolysaccharidosis type I (MPS I) and type II (MPS II), respectively; CHAMPIONS: A Phase 1/2 clinical trial with dose escalation of SB-913 ZFN-mediated in vivo human genome editing for treatment of MPS II (Hunter syndrome); and EMPOWERS: A Phase 1/2 clinical trial of SB-318 ZFN-mediated in vivo human genome editing for treatment of MPS I (Hurler Syndrome). The SB-913 (MPS II) presentation is expected to include interim data on safety and biochemical measurements at up to 24 weeks from six subjects enrolled in the three dose cohorts of the CHAMPIONS Study. The SB-318 (MPS I) presentation is expected to describe the scientific rationale for SB-318, the clinical trial design, and preliminary safety and biochemical measurements at up to four weeks from the first three patients enrolled in the EMPOWERS Study. Sangamo expects to provide additional SB-913 and SB-318 updates in 2019 as data accumulate and mature in these clinical development programs.
Sangamo Therapeutics, Inc. is a genomic medicines company that is developing medicines for neurological diseases. The Company's neurology preclinical development is focused on two areas: development of epigenetic regulation therapies to treat serious neurological diseases, and development of novel engineered adeno-associated virus (AAV) capsids to deliver its therapies to the intended neurological targets. The Company's zinc finger epigenetic regulators are ideally suited to potentially address neurological disorders and its capsid discovery platform is expanding delivery beyond intrathecal delivery capsids, including in the central nervous system. Its clinical-stage product candidates are Isaralgagene civaparvovec, also known as ST-920, its wholly-owned gene therapy product candidate for the treatment of Fabry disease, and TX200, its wholly-owned CAR-Treg cell therapy product candidate for the prevention of immune-mediated rejection in HLA-A2 mismatched kidney transplantation.