Sangamo Therapeutics

Ionis Pharmaceuticals' Tryngolza is the first FDA-approved treatment for familial chylomicronemia syndrome (FCS). FDA senior advisor Robert Temple to retire. Zealand Pharma's glepaglutide for short bowel syndrome rejected, requiring an additional trial. Sangamo grants Astellas rights to use a viral capsid for gene therapy delivery. BioAge Labs partners with Novartis to discover drug targets for age-related illnesses.

Sangamo Therapeutics and Astellas Pharma entered a license agreement for Sangamo's neurotropic AAV capsid, STAC-BBB, granting Astellas exclusive rights for one target with options for four additional neurological disease targets. Sangamo will receive a $20 million upfront fee and could earn up to $1.3 billion in additional target fees and milestone payments, plus royalties on potential net sales. Astellas will manage all research, development, manufacturing, and commercialization activities.

Sangamo Therapeutics' ST-503, an epigenetic therapy targeting SCN9A gene for idiopathic small fiber neuropathy, received FDA IND clearance. The Phase I/II trial in mid-2025 aims to assess safety and efficacy, offering hope for chronic neuropathic pain treatment.

The global genome editing market, valued at USD 5.5 billion in 2022, is projected to grow at a CAGR of 13.9% to USD 17.5 billion by 2031, driven by advancements in CRISPR and other gene-editing technologies, increasing applications in therapeutics and agriculture, and rising investments in genetic research and development.

DelveInsight's report on Alzheimer's Disease pipeline highlights 110+ companies developing 120+ therapies, including key players like Neurodon, Sangamo Therapeutics, and Eisai Co Ltd. Notable therapies in clinical trials are NRDN-201, ST-501, and Simufilam. Recent developments include FDA approval of Leqembi and ongoing Phase 1 trials for ML-007/PAC by MapLight Therapeutics. The report covers pre-clinical to marketed phases, detailing drug MOA, ROA, and market dynamics.

The mRNA Therapeutics Market was valued at USD 11.82 billion in 2023 and is projected to reach USD 48.65 billion by 2032, with a CAGR of 17.04%. The market's growth is driven by mRNA-based vaccines against COVID-19, advancements in mRNA-based cancer therapies, and regulatory approvals. North America leads the market, followed by Europe, while Asia Pacific is expected to grow the fastest.

The gene editing market is projected to grow from USD 4.66 billion in 2024 to USD 7.59 billion by 2029, driven by advancements in technologies and expanding applications. The products segment, including kits, reagents, libraries, and software, dominates the market. CRISPR technology leads, while Asia Pacific shows the highest growth rate due to increased R&D and government funding. Challenges include off-target effects and scalability issues.

The Gene Switch Market is projected to grow from USD 0.78 Bn in 2024 to USD 1.78 Bn by 2031, with a CAGR of 11.6%. CoherentMI's report provides a comprehensive analysis of market trends, competitive landscape, and key players like Novartis, Pfizer, and Gilead Sciences.

DelveInsight's 'Fabry Disease Pipeline Insight, 2024' report details 18+ companies developing 18+ therapies, including Venglustat, Pegunigalsidase alfa, and 4D 310, with insights on mechanism of action, route of administration, and clinical trials. Key companies include Idorsia Pharmaceuticals, Protalix, Sanofi Genzyme, and Sangamo Therapeutics. The report covers pre-clinical to marketed phases, focusing on commercial and clinical assessments.

Sangamo Therapeutics received FDA clearance for its IND application for ST-503, an epigenetic regulator targeting chronic neuropathic pain in idiopathic small fiber neuropathy (iSFN), with plans to initiate a Phase 1/2 study in mid-2025. ST-503 uses AAV vector technology with zinc finger repressors to target the SCN9A gene, addressing an estimated 43,000 U.S. patients with iSFN and broader peripheral neuropathies impacting nearly 40 million Americans.