SANGAMO THERAPEUTICS, INC

SANGAMO THERAPEUTICS, INC is a genomic medicine company focused on developing medicines for serious neurological diseases. The company utilizes zinc finger epigenetic regulators and a capsid engineering platform. Its core neurology programs concentrate on two areas: developing epigenetic regulation therapies for neurological diseases and creating novel engineered adeno-associated virus (AAV) capsids to deliver these therapies to neurological targets. Initial wholly-owned preclinical programs target small fiber neuropathy (SFN), a type of chronic neuropathic pain, and prion disease. In November 2024, the U.S. Food and Drug Administration (FDA) cleared the investigational new drug application (IND) for ST-503 for SFN. Following this, the first six Phase 1/2 clinical sites have been activated, and patient recruitment and enrollment have commenced. The company is also preparing a clinical trial application (CTA) for its prion disease product candidate. SANGAMO THERAPEUTICS, INC collaborates with partners such as Genentech, Inc. (a member of the Roche Group), Astellas Gene Therapies, Inc., Alexion Pharmaceuticals, Inc., Eli Lilly and Company, and Takeda Pharmaceutical Company Limited. These collaborations involve the development of preclinical product candidates for indications including tauopathies, amyotrophic lateral sclerosis (ALS), and other undisclosed neurology targets. The company is also developing novel engineered AAV capsids for neurological delivery. It has identified a proprietary engineered neurotropic AAV capsid variant, STAC-BBB, which has demonstrated the ability to cross the blood-brain barrier (BBB) in nonhuman primates (NHPs) and mice. STAC-BBB mediated robust transduction, transgene expression, and targeted epigenetic repression throughout the brain and spinal cord after intravenous (IV) administration.