Sangamo Therapeutics

In October 2024, the FDA cleared IND applications for an allogeneic CAR-engineered gamma delta T-cell therapy for IIM and SPS, the first IND clearance for a circular RNA therapy, and held meetings with companies planning BLA submissions for advanced therapeutics. Nanoscope plans to file a BLA for RP gene therapy MCO-010 in Q1 2025. Adicet's ADI-100 received IND amendment clearance for IIM and SPS trials. Sangamo aligned with FDA on an accelerated approval pathway for Fabry disease gene therapy ST-920. RiboX's RXRG001, the first circular RNA therapy, was cleared for a phase 1/2a trial in RIX and hyposalivation.

Sangamo Therapeutics' shares surged 69% after FDA alignment on an Accelerated Approval pathway for its Fabry disease gene therapy, isaralgagene civaparvovec (ST-920), using data from its Phase I/II STAAR trial. The company plans to submit a BLA in the second half of 2025, three years ahead of previous estimates.

Data from the Phase 1/2 STAAR study of ST-920, an investigational gene therapy for Fabry disease, could support its accelerated approval by the FDA, potentially allowing Sangamo Therapeutics to submit a biologics license application in 2025, three years ahead of schedule. ST-920 aims to deliver a healthy version of the GLA gene to liver cells, enabling the production of a functional alpha-Gal A enzyme. Interim data from the study showed ST-920 was well-tolerated, with sustained increases in alpha-Gal A levels and reduced symptoms. The FDA agreed that the eGFR slope at 52 weeks could serve as a primary basis for approval, with a calculation at 104 weeks suggested for long-term benefit confirmation.

At the ESGCT annual meeting in Rome, inclement weather disrupted travel. Jeremy Duffield, CSO of Prime Medicine, criticized Tessera Therapeutics' gene editing technology, emphasizing Prime's established prime editing. Michael Holmes, Tessera's CSO, presented in vivo RNA Gene Writer data for sickle cell disease and α-1 antitrypsin deficiency, aiming for one-shot therapies. Duffield later showcased Prime's preclinical data on liver diseases, highlighting LNP delivery and editing efficiency.

Sangamo Therapeutics plans to use 1-year posttreatment eGFR slope data from its phase 1/2 STAAR trial for an accelerated approval pathway for ST-920, a gene therapy for Fabry disease, with a BLA submission expected in 2025. The therapy showed statistically significant improvements in eGFR levels and potential to alleviate Fabry disease symptoms, offering a one-time treatment alternative to enzyme replacement therapy.

Sangamo Therapeutics announced a successful interaction with the FDA, paving the way for Accelerated Approval of its gene therapy ST-920 for Fabry disease. The FDA agreed that data from the Phase 1/2 STAAR study can support approval using eGFR slope at 52 weeks as an intermediate clinical endpoint, with a potential BLA submission anticipated in the second half of 2025.

Sangamo Therapeutics aligns with FDA for Accelerated Approval of ST-920 in Fabry disease, with BLA submission expected in 2025, using eGFR slope at 52 weeks as an intermediate clinical endpoint.

Pfizer's Hympavzi, a once-weekly injection for hemophilia A or B, received FDA approval. The drug, administered via auto-injector, aims to reduce bleeding episodes and treatment burden. Sales are projected to reach $300 million by 2030.