SANGAMO THERAPEUTICS, INC.

We are a genomic medicine company focused on developing medicines for serious neurological diseases. Our work involves two main areas: epigenetic regulation therapies and novel engineered adeno-associated virus (AAV) capsids for targeted delivery. Our wholly owned preclinical programs are initially focused on 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 our investigational new drug application (IND) for ST-503 for SFN, and we have activated the first six Phase 1/2 clinical sites and started patient recruitment and enrollment. We are also preparing a clinical trial application (CTA) for our prion disease product candidate. We collaborate with partners like Genentech, Inc., Astellas Gene Therapies, Inc., Alexion Pharmaceuticals, Inc., Eli Lilly and Company, and Takeda Pharmaceutical Company Limited, who are developing preclinical product candidates for indications such as tauopathies, amyotrophic lateral sclerosis (ALS), and other undisclosed neurology targets. We are also developing novel engineered AAV capsids for neurological targets, including STAC-BBB, a proprietary neurotropic AAV capsid variant. STAC-BBB has shown the ability to cross the blood-brain barrier (BBB) in nonhuman primates (NHPs) and mice, and has mediated robust transduction, transgene expression, and targeted epigenetic repression throughout the brain and spinal cord after intravenous (IV) administration.