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.
相关临床试验
28
3 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
N/A
进行中（未招募）
3
10.7%
已完成
19
67.9%
招募中
1
3.6%
终止
5
17.9%
暂无批准数据
- PTC Therapeutics was selected as the winning bidder to acquire ST-920, a BLA-stage one-time AAV gene therapy for Fabry disease, from Sangamo Therapeutics in a competitive bankruptcy auction. - The transaction includes $111 million upfront and up to $100 million in contingent milestone payments, with a rolling BLA submission to the FDA expected to be completed in Q4 2026. - The Phase 1/2 STAAR study demonstrated a positive mean annualized eGFR slope at 52 weeks and sustained α-Gal A activity for up to 4.5 years, with no requirement for routine immunosuppressive agents. - ST-920 holds Orphan Drug, Fast Track, and RMAT designations from the FDA, with potential commercial launch anticipated in 2027.
- The global gene editing therapeutics market is projected to grow at a compound annual growth rate of 12.5%, fueled by expanding clinical applications and rising disease burden. - CRISPR-based technologies dominate the market due to their efficiency and cost-effectiveness, while oncology represents the largest therapeutic application segment. - High development costs, regulatory complexity, and off-target safety concerns remain significant barriers to broader adoption and commercialization. - Key players including CRISPR Therapeutics, Intellia Therapeutics, Editas Medicine, and Beam Therapeutics are driving innovation through partnerships and investments in next-generation editing platforms.
- Sangamo Therapeutics, a three-decade-old gene editing pioneer, filed for Chapter 11 bankruptcy with $115 million in debt and just $5.5 million in cash remaining. - Eli Lilly has offered $50 million for Sangamo's core gene editing platforms and an experimental prion disease treatment, while Astellas bid up to $50 million for a Fabry disease gene therapy nearing regulatory approval. - The company's partnership model collapsed after Novartis, Biogen, and Pfizer terminated major collaborations, causing revenues to plummet from $176.2 million in 2023 to $39.6 million in 2025. - A court-supervised auction is scheduled for next month, with 77 employees retained specifically for the asset purchases by Lilly and Astellas.
- Sangamo Therapeutics has filed for Chapter 11 bankruptcy protection in a strategic move to facilitate the sale of the company. - The gene therapy developer received initial court approval for $30 million in debtor-in-possession (DIP) financing to support operations during the restructuring process. - The bankruptcy filing and DIP financing are designed to preserve the company's gene therapy pipeline and platform while pursuing a going-concern sale. - The outcome of the sale process will determine the future of Sangamo's genomic medicine programs and its proprietary zinc finger nuclease technology.
- Sangamo Therapeutics' gene therapy ST-920 (isaralgagene civaparvovec) is advancing toward accelerated FDA approval with a BLA submission planned for the latter half of 2025, following positive Phase 1/2 STAAR trial data. - The global Fabry disease treatment market was valued at USD 2.62 billion in 2025 and is projected to reach USD 5.92 billion by 2035, growing at a CAGR of 8.47%. - Enzyme replacement therapy remains the standard of care with 76.10% market share in 2025, while gene therapies and oral chaperone treatments are expected to reshape the treatment landscape. - In 2024, approximately 9,200 diagnosed prevalent cases of Fabry disease were estimated in the United States, representing 52% of the total across the seven major markets.
- Tango Therapeutics appointed Dr. Malte Peters as President and CEO, replacing founding CEO Dr. Barbara Weber who transitions to Executive Chair. - The leadership change comes as the company's lead PRMT5 inhibitor vopimetostat prepares to enter registrational trials for pancreatic cancer in 2026. - Dr. Peters brings extensive late-stage clinical development experience, having previously led global regulatory approval of Monjuvi at MorphoSys AG. - The company maintains its 2026 clinical milestone guidance, including combination trial data and pivotal study initiation for vopimetostat.
- Regulatory T cell (Treg) therapies are rapidly expanding beyond polyclonal approaches to include engineered CAR-Treg and TCR-Treg products, with 69 clinical trials underway as of 2025 targeting autoimmune diseases and transplant rejection. - Advanced monitoring technologies including single-cell sequencing, spatial omics, and deuterium labeling are enabling comprehensive tracking of Treg persistence, stability, and function in clinical trials. - Regulatory guidance for Treg therapies relies on risk-based approaches leveraging existing frameworks for cell and gene therapies, with emphasis on in vitro efficacy studies and target liability assessments due to limited appropriate preclinical models. - Clinical observations from CAR-T therapy trials have identified CAR-expressing Tregs as negative correlates of patient outcomes, providing evidence for engineered Treg function in humans and informing monitoring strategies.
- Sangamo's isaralgagene civaparvovec demonstrated positive kidney function outcomes in Fabry disease patients, with a mean annualized eGFR slope of 1.965 mL/min/1.73m²/year at 52 weeks across 32 dosed patients. - The FDA reiterated its agreement to use eGFR slope as an endpoint for accelerated approval pathway, positioning the company for a potential BLA submission in Q1 2026. - All 18 patients who began the study on enzyme replacement therapy were successfully withdrawn from ERT and remained off treatment, demonstrating the therapy's potential as a durable alternative. - The company also initiated patient enrollment in its Phase 1/2 STAND study for chronic neuropathic pain treatment ST-503, marking its first neurology clinical trial.
- Roche has entered a partnership with Boston-based Manifold Bio, providing $55 million upfront with potential total value exceeding $2 billion to develop AI-driven brain drug delivery technologies. - Manifold's platform uses artificial intelligence to identify thousands of "shuttles" that can cross the blood-brain barrier, testing drug candidates in living organisms rather than traditional petri dish methods. - The collaboration targets neurological and neurodegenerative diseases including Alzheimer's and Parkinson's, with Roche maintaining 21 neuroscience programs currently in human trials. - Manifold retains rights to apply its shuttle technology to other therapeutic areas while Roche gains access to specific programs for brain drug delivery applications.
- Over 180 companies are actively developing more than 250 AAV-based gene therapies, representing a robust pipeline for treating genetic disorders including hemophilia, spinal muscular atrophy, and inherited retinal diseases. - Recent FDA approvals include Roctavian for severe hemophilia A and Elevidys for Duchenne muscular dystrophy, demonstrating the therapeutic potential of AAV vectors in addressing rare genetic conditions. - The field has experienced notable setbacks, including Pfizer's termination of its hemophilia A partnership and a patient death linked to Elevidys treatment, highlighting ongoing safety challenges. - Leading pipeline candidates include DTX401 for glycogen storage disease, SRP-9003 for limb-girdle muscular dystrophy, and several innovative therapies targeting retinal diseases and cardiovascular conditions.
