Sangamo Therapeutics

Global Acquired Hemophilia A pipeline comprises over 10 companies developing innovative therapies across various clinical stages, according to DelveInsight's 2025 report.

• Sangamo will present data on its novel zinc finger repressor therapy for prion disease at the ASGCT Presidential Symposium, demonstrating sustained brain-wide protein suppression in mice and non-human primates. • The company's innovative approach combines zinc finger epigenetic regulators with its proprietary STAC-BBB delivery system, potentially offering a one-time intravenous treatment for this currently incurable neurodegenerative disease. • Additional presentations will highlight Sangamo's advances in chronic pain treatment targeting Nav1.7, second-generation AAV capsid engineering, and manufacturing innovations for CNS delivery systems.

Eli Lilly has secured rights to Sangamo Therapeutics' STAC-BBB capsid technology for $18 million upfront, with potential milestone payments reaching $1.4 billion plus tiered royalties.

Sangamo Therapeutics has secured FDA clearance for its first neurology IND targeting idiopathic small fiber neuropathy, with patient enrollment expected to begin in mid-2025 and preliminary efficacy data anticipated by Q4 2026.

Pfizer has announced the cessation of development and commercialization of Beqvez, its FDA-approved gene therapy for hemophilia B, citing limited interest from patients and healthcare providers.

Updated Phase 1/2 STAAR study data demonstrates sustained α-Gal A enzyme activity for up to 47 months, with all 18 ERT-dependent patients successfully discontinuing enzyme replacement therapy.

• Pfizer has terminated its partnership with Sangamo Therapeutics for the hemophilia A gene therapy, giroctocogene fitelparvovec, despite promising Phase III trial results. • This decision by Pfizer casts doubt on the commercial viability of first-generation hemophilia A gene therapies, influenced by the slow market uptake of BioMarin's Roctavian. • Sangamo's stock plummeted by 56% following the announcement, as the company loses potential milestone payments of up to $220 million from Pfizer. • Sangamo now intends to explore all options for advancing the gene therapy, including seeking a new collaboration partner, while facing financial challenges with limited cash reserves.

• Pfizer has decided to discontinue development of giroctocogene fitelparvovec for hemophilia A, returning full rights to Sangamo Therapeutics despite positive Phase 3 trial results. • The decision marks another strategic retreat from gene therapy by Pfizer, though the company maintains its hemophilia B treatment Beqvez in its portfolio. • Sangamo Therapeutics, expressing surprise and disappointment, is now seeking new partnership opportunities to advance the promising gene therapy program.

• Over 120 Alzheimer's Disease treatment therapies are under development by 110+ companies globally, ranging from preclinical to marketed phases. • Emerging therapies like NRDN-201, ST-501, and KarXT are in various clinical trial phases, showing potential for significant market impact. • MapLight Therapeutics initiated a Phase 1 trial for ML-007/PAC, targeting schizophrenia and Alzheimer's disease psychosis, with Phase 2 trials planned. • The FDA granted conventional approval to Leqembi (lecanemab-irmb), marking the first amyloid beta-directed antibody to transition from accelerated approval.

• Clinical trials are actively progressing for Fabry Disease, with companies like Idorsia and Sanofi Genzyme developing novel therapies. • Emerging treatments like Venglustat and Pegunigalsidase alfa are expected to significantly impact the Fabry Disease market in the coming years. • Research and development efforts are also focused on Clostridium Difficile Infections, with Vedanta Biosciences leading Phase 3 trials for VE303. • Mydriasis treatments are advancing, with companies like Eyenovia exploring solutions like MYDCOMBI for pupil dilation.