Stoke Therapeutics, Inc. is a biotechnology company, which engages in the research and development of treatments for genetic diseases. It offers a wide range of relevant tissues including the central nervous system, eye, kidney, and liver. The company was founded by Isabel Aznarez and Adrian R. Krainer in June 2014 and is headquartered in Bedford, MA.
相关临床试验
6
3 进行中
药物批准
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监管机构
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监管机构数
成立时间
2014
进行中（未招募）
2
33.3%
已完成
2
33.3%
尚未招募
1
16.7%
招募中
1
16.7%
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- Encoded Therapeutics closed a $275 million Series F financing co-led by GV and an undisclosed healthcare fund, with participation from ARCH Venture Partners, SoftBank Vision Fund 2 and others. - Proceeds will fund a pivotal trial of ETX101 in infants and young children with SCN1A-positive Dravet syndrome plus an expansion study up to age 18. - Interim Phase 1/2 POLARIS data presented at the European Epilepsy Congress showed up to 79% reductions in monthly seizures at the third dose level after one year. - Funding also supports internal GMP manufacturing scale-up and advancement of ETX301 toward a 2027 IND submission for post-amputation neuroma pain.
- The developmental and epileptic encephalopathies (DEE) market reached USD 2.2 billion across the 7MM in 2025, with the United States accounting for approximately 59% of the total market share. - The diagnosed prevalent population of DEE in the 7MM is projected to rise from approximately 288,000 in 2025 to 326,000 by 2036, reflecting improved genetic testing and diagnostic awareness. - A robust pipeline featuring antisense oligonucleotides, gene therapies, and precision medicines—including zorevunersen, bexicaserin, and relutrigine—is expected to reshape the treatment landscape for genetically defined DEE subtypes. - Praxis Precision Medicines' relutrigine received FDA Priority Review acceptance in March 2026 with a PDUFA target action date of September 27, 2026, for SCN2A and SCN8A DEEs.
- A new experimental drug called zorevunersen demonstrated up to 91% reduction in seizures among children with Dravet syndrome in an international clinical trial led by UCL and Great Ormond Street Hospital. - The gene therapy treatment works by increasing protein levels from the healthy SCN1A gene copy to restore proper nerve cell function in patients with this devastating genetic epilepsy condition. - Results from 81 children aged 2-18 showed the drug is safe and well-tolerated, with patients experiencing improved quality of life and cognitive function over a three-year period. - A Phase Three study is currently underway to further evaluate the treatment's effectiveness before potential regulatory approval.
- NervGen Pharma appointed Shamim Ruff as Chief Regulatory Affairs Officer and Christine McSherry as SVP of Patient Advocacy and Clinical Affairs to advance their spinal cord injury treatment program. - Ruff brings over 30 years of regulatory expertise from companies including Stoke Therapeutics and Sarepta Therapeutics to guide NVG-291 toward potential approval as the first pharmacologic treatment for SCI. - McSherry, who founded the Jett Foundation after her son's Duchenne muscular dystrophy diagnosis, will ensure the SCI community's voice remains central to NervGen's clinical strategy. - The appointments strengthen NervGen's leadership as the company prepares for Phase 3 trials of NVG-291, which demonstrated durable improvements in function and quality of life in chronic SCI patients.
- Stoke Therapeutics has dosed the first patient in the Phase 1 OSPREY study of STK-002, an antisense oligonucleotide designed to treat Autosomal Dominant Optic Atrophy (ADOA). - ADOA is the most common inherited optic nerve disorder affecting approximately one in 30,000 people globally, causing progressive vision loss with no currently approved treatments. - STK-002 aims to restore OPA1 protein expression by targeting the underlying genetic cause of ADOA, potentially becoming the first disease-modifying therapy for this condition. - The dose-escalating study will evaluate safety and tolerability in patients aged 6-55 with confirmed OPA1 gene variants, with recruitment active in the UK and Germany.
- Stoke Therapeutics surged 11.42% after presenting positive data for zorevunersen in Dravet syndrome at the 2025 AES Annual Meeting with partner Biogen. - Alpha Tau Medical climbed 25.26% following announcement that the first patient was treated in its pilot study for recurrent glioblastoma multiforme using Alpha DaRT technology. - Senti Biosciences gained 3.92% after the FDA granted RMAT designation to SENTI-202, an investigational CAR-NK cell therapy for acute myeloid leukemia and other hematologic malignancies. - Multiple biotech companies showed strong after-hours performance driven by clinical data presentations, regulatory progress, and financing announcements across various therapeutic areas.
- Chinese regulators have accepted a Biologics License Application for LEQEMBI's subcutaneous formulation, marking a significant regulatory milestone for Biogen and partner Eisai. - Clinical data from CTAD 2025 demonstrates that the subcutaneous version maintains comparable exposure and safety profiles to the intravenous formulation while offering greater treatment convenience. - The development reinforces Biogen's strategy to establish disease-modifying therapies in high-need neurological conditions, particularly Alzheimer's disease treatment. - Recent partnerships with Stoke Therapeutics and Dayra Therapeutics expand Biogen's neurology pipeline beyond Alzheimer's to include Dravet syndrome and oral macrocyclic peptide research.
- Stoke Therapeutics reports strong progress in its Phase 3 EMPEROR study for zorevunersen in Dravet syndrome, with more than 20 patients randomized and approximately 35 additional patients in screening as of October 2025. - The company maintains a robust financial position with $328.6 million in cash and securities as of September 30, 2025, providing funding runway through mid-2028 to support operations and commercialization preparations. - Recent clinical data presentations demonstrate zorevunersen's potential for disease modification, showing continuing improvements in cognition and behavior at two years, contrasting with minimal changes in standard-of-care treated patients. - The company has initiated Phase 1 OSPREY study for STK-002 in Autosomal Dominant Optic Atrophy and plans to identify a clinical candidate for SYNGAP1 treatment in 2026.
- Stoke Therapeutics announced new results from the ongoing Phase 3 EMPEROR study of zorevunersen (STK-001) for Dravet syndrome, a rare and severe form of epilepsy. - The company published final results from the two-year BUTTERFLY natural history study highlighting persistent developmental and seizure challenges in Dravet syndrome patients despite current treatments. - The BUTTERFLY study validates the clinical endpoints used in the pivotal EMPEROR Phase 3 trial, underscoring the urgent unmet medical need for disease-modifying therapies. - The research reinforces zorevunersen's potential as a first-in-class therapy for addressing Dravet syndrome's longstanding therapeutic gap.
- Stoke Therapeutics presented two-year data from the FALCON study, the largest prospective natural history study of autosomal dominant optic atrophy (ADOA), enrolling 47 patients across 10 international sites. - The study found that while ADOA progresses slowly, 24% of patients experienced at least a five-letter loss in low-contrast visual acuity, and patients showed higher levels of mitochondrial dysfunction compared to healthy individuals. - No significant anatomic changes in the retina were observed, suggesting retinal dysfunction may be reversible with treatment intervention, supporting development of STK-002 as a potential disease-modifying therapy. - The data inform the ongoing Phase 1 OSPREY study of STK-002, an antisense oligonucleotide designed to upregulate OPA1 protein expression and potentially become the first approved treatment for ADOA.
