Sangamo Therapeutics
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- -
- Employees
- 405
- Market Cap
- $170.2M
- Introduction
Sangamo Therapeutics, Inc. is a clinical stage biotechnology company, which engages in the research and development of zinc finger proteins. It focuses on three therapeutic areas: inherited metabolic diseases, central nervous system, and diseases and immunology, which consist of inflammatory and autoimmune diseases. It also offers complementary technology platforms such as gene therapy, ex vivo cell therapy, in vivo genome editing, and in vivo genome regulation. The company was founded by Edward O. Lanphier II in 1995 and is headquartered in Richmond, CA.
Acquired Hemophilia A Pipeline Expands with 10+ Novel Therapies in Development
• Global Acquired Hemophilia A pipeline comprises over 10 companies developing innovative therapies across various clinical stages, according to DelveInsight's 2025 report.
• Recent clinical advances include promising results for rituximab as a potential first-line therapy and Pfizer's positive Phase 3 AFFINE trial results for giroctocogene fitelparvovec gene therapy.
• Key pharmaceutical players including Novo Nordisk, Sanofi, Pfizer, and Belief Biomed are advancing treatments ranging from gene therapies to monoclonal antibodies targeting this rare autoimmune bleeding disorder.
Eli Lilly Partners with Rznomics in $1.3B Deal to Develop RNA Editing Therapy for Hereditary Hearing Loss
• Rznomics Inc. has secured a potential $1.35 billion global license option agreement with Eli Lilly to codevelop an RNA editing-based gene therapy targeting hereditary hearing loss.
• Under the partnership structure, Rznomics will lead initial R&D efforts to discover and develop the therapy, while Lilly will handle subsequent development and commercialization phases.
• The deal represents a significant expansion of Lilly's gene therapy pipeline for hearing disorders, though specific details regarding up-front payments and option terms remain undisclosed.
Stylus Medicine Emerges with $85M to Revolutionize In Vivo Genetic Medicines
• Stylus Medicine has emerged from stealth with $85 million in financing to develop its platform combining sequence-specific genome integration with cell-targeted lipid nanoparticle delivery.
• The company's technology aims to overcome limitations of ex vivo cell therapy manufacturing by enabling durable, flexible, and scalable in vivo cell engineering, with initial focus on CAR-T therapies.
• Led by industry veterans Emile Nuwaysir (CEO) and Jason Fontenot (CSO), Stylus has secured backing from prominent investors including RA Capital Management, Khosla Ventures, Chugai Venture Fund, Eli Lilly, and Johnson & Johnson Innovation.
Vertex Pharmaceuticals Abandons AAV Vector Technology Amid Broader Industry Retreat from Gene Therapy
• Vertex Pharmaceuticals has discontinued all research on adeno-associated virus (AAV) vector technology, impacting partnerships with Affinia Therapeutics and Tevard Biosciences focused on Duchenne muscular dystrophy treatments.
• The retreat from AAV vectors follows a broader industry trend, with Pfizer, Roche, Takeda, and Biogen all scaling back gene therapy programs due to safety concerns, limited payload capacity, and high manufacturing costs.
• Despite industry pullback, companies like Affinia Therapeutics continue developing next-generation AAV vectors, while others explore alternative delivery systems such as Herpes simplex virus-1 vectors with larger genetic payloads.
Sangamo Therapeutics to Showcase Breakthrough Prion Disease Treatment at ASGCT Annual Meeting
• 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.
Lilly Licenses Sangamo's Blood-Brain Barrier Capsid Technology in $1.4B CNS Deal
• 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.
• The agreement allows Lilly to utilize Sangamo's proprietary adeno-associated virus capsid that can cross the blood-brain barrier to deliver genomic medicines targeting central nervous system diseases.
• This partnership provides critical financial support for Sangamo, which had only enough cash to operate into Q1 2025 following Pfizer's termination of their hemophilia gene therapy collaboration.
B-Cell Lymphoma Pipeline Expands with 300+ Therapies in Development for 2025
• DelveInsight's latest report reveals a robust B-cell lymphoma pipeline with over 295 companies developing 300+ therapies, highlighting significant industry investment in this area.
• Several major pharmaceutical companies including BeiGene, Celgene, Hoffmann-La Roche, and Allogene Therapeutics have initiated pivotal late-stage clinical trials for novel B-cell lymphoma treatments in March 2025.
• Emerging therapies include CAR-T cell approaches, bispecific antibodies, and novel targeted agents, with many incorporating dual-targeting mechanisms to overcome resistance seen with single-target therapies.
Sangamo Therapeutics Advances Neurology Pipeline and Fabry Disease Gene Therapy Amid Financial Restructuring
• 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.
• The company's Fabry disease gene therapy has demonstrated sustained benefits with a positive mean eGFR slope of 3.061 mL/min/1.73m² in 23 patients, with all 18 patients who began on enzyme replacement therapy successfully withdrawn and remaining off treatment.
• Despite reducing non-GAAP operating expenses by nearly half year-over-year and raising over $100 million in 2024, Sangamo faces financial challenges with cash runway extending only into mid-Q2 2025 as it seeks commercialization partners for key programs.
Biotech Deal Landscape: February-March 2025 Sees Surge in Partnerships Across Multiple Therapeutic Areas
• The first quarter of 2025 witnessed significant biotech partnership activity, with Eli Lilly, AstraZeneca, and Novo Nordisk emerging as top collaborators in deals worth billions across small molecules, antibodies, and RNA therapeutics.
• February 2025 featured notable acquisitions including Novartis's $2.15 billion buyout of Anthos Therapeutics, while March saw AstraZeneca acquire Belgian biotech EsoBiotec and Bristol Myers Squibb purchase 2seventy bio for $286 million.
• Obesity therapeutics gained significant traction in March 2025, with AbbVie entering the field through a $350 million upfront deal with Gubra for an amylin analog, while Roche partnered with Zealand Pharma on petrelintide in a deal worth up to $5.25 billion.
Praxis Therapeutics' Essential Tremor Drug Faces Setback in Phase 3 Interim Analysis
• Praxis Therapeutics announced that their lead drug candidate for essential tremor failed to meet primary endpoints in an interim Phase 3 analysis, marking a significant setback for the company's development program.
• Despite the disappointing interim results, the company has decided to continue the Phase 3 trial to completion, citing the need for comprehensive data analysis and potential insights from the full study population.
• Essential tremor remains a challenging neurological condition with limited treatment options, highlighting the continued unmet medical need in this therapeutic area.
Pfizer Discontinues Beqvez Gene Therapy for Hemophilia B Amid Low Market Demand
• 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.
• The decision marks Pfizer's complete exit from viral gene replacement therapies, following earlier withdrawals from Duchenne muscular dystrophy and hemophilia A programs.
• The company will shift focus to other treatments including Hympavzi for hemophilia A, while maintaining support for existing Beqvez clinical trial participants.
Amneal Launches Generic Ezetimibe, Competing with $2.7B Zetia Market
• Amneal Pharmaceuticals has introduced generic ezetimibe tablets, a direct competitor to Merck's cholesterol-lowering drug Zetia, in 10mg strength across multiple bottle sizes.
• The generic launch targets a substantial market opportunity, with Zetia recording approximately $2.7 billion in annual U.S. sales as of April 2017.
• Distribution has commenced from Amneal's Kentucky facility, offering healthcare providers and patients a more affordable alternative to the branded medication.
Sangamo's Gene Therapy Shows Sustained Benefits in Fabry Disease Patients with Improved Kidney Function
• 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.
• Patients showed significant kidney function improvements with a positive mean annualized eGFR slope of 3.061 mL/min/1.73m2/year, indicating potential disease-modifying effects.
• Sangamo plans BLA submission in second half of 2025 following FDA's agreement to consider Accelerated Approval pathway based on 52-week eGFR data.
Astellas Seeks First Japanese Approval for Geographic Atrophy Treatment with Avacincaptad Pegol
• Astellas Pharma has submitted a New Drug Application to Japan's MHLW for avacincaptad pegol, potentially becoming the first approved treatment for geographic atrophy in Japan.
• The submission is supported by GATHER1 and GATHER2 clinical trials, which demonstrated the drug's ability to slow GA lesion growth with a favorable safety profile over two years.
• Geographic atrophy affects over 5 million people globally, with 66% at risk of becoming legally blind without treatment, highlighting the significant unmet medical need.
Telethon Foundation Seeks EMA Approval for Etuvetidigene Autotemcel in Wiskott-Aldrich Syndrome
• Telethon Foundation has submitted a marketing authorization application to the EMA for etuvetidigene autotemcel, a gene therapy for Wiskott-Aldrich syndrome.
• Etuvetidigene autotemcel is a one-time treatment for patients lacking a matched family donor for hematopoietic stem cell transplantation.
• The therapy has been administered to 30 patients, with availability in Italy since August 2023 for patients over six months without a matched related donor.
• Telethon is also preparing a submission to the FDA to make the therapy available in the US, marking their second venture into taking over gene therapy development.
Gene Therapy Landscape Expands for Fabry Disease: Multiple Clinical Trials Show Promise in 2024
Multiple gene therapy trials for Fabry disease demonstrated significant progress in 2024, with AMT-191, 4D-310, and ST-920 showing promising safety and efficacy data. These therapies aim to address the underlying genetic cause of Fabry disease through different approaches, potentially offering long-term treatment options for patients.
2024 Breakthroughs and Setbacks in Lysosomal Disorder Gene Therapies
Major developments in lysosomal disorder treatments marked 2024, with both advances and challenges in gene therapy approaches. Notable events included Roche's discontinuation of its Pompe disease program, promising progress in Fabry disease treatment by Sangamo, and mixed results in other rare disease therapies.
Pfizer Abandons Hemophilia A Gene Therapy, Jeopardizing Sangamo's Financial Stability
• 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.
FDA Approves Journavx, a Novel Non-Opioid Painkiller, Offering New Hope for Acute Pain Management
• The FDA has approved Journavx (suzetrigine), a first-in-class non-opioid analgesic, for treating moderate to severe acute pain in adults, offering a safer alternative to opioids.
• Journavx works by selectively blocking pain signals in the peripheral nervous system, reducing pain without the addictive potential and severe side effects associated with opioids.
• Clinical trials demonstrated Journavx's effectiveness in reducing pain after surgeries, with a safety profile comparable to placebo, though it didn't outperform opioid-acetaminophen combinations.
• Priced at $15.50 per pill, Journavx faces challenges in accessibility due to its higher cost compared to generic opioids, but it represents a significant step in combating the opioid crisis.
GLP-1 Receptor Agonists Show Promise in Alzheimer's Disease: Key Clinical Trial Readouts Expected in 2025
• Phase III trial results for semaglutide (EVOKE and EVOKE Plus) are anticipated in September 2025, investigating its potential to slow cognitive decline in Alzheimer's patients.
• Studies suggest GLP-1 drugs like semaglutide and liraglutide may reduce Alzheimer's risk and slow cognitive decline, possibly by improving insulin resistance in the brain.
• Experts emphasize the need for large, controlled trials to confirm GLP-1s' efficacy and safety for chronic use in preventing or treating Alzheimer's disease.
• Researchers are exploring GLP-1/GIP receptor agonists like Kariya's KP405, designed to better penetrate the brain, potentially minimizing side effects associated with GLP-1 treatments.
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