Voyager Therapeutics, Inc. is a clinical-stage gene therapy company, which engages in the development of treatments for severe neurological diseases. Its pipeline of gene therapy programs includes VY-AADC, VY-SOD101, VY-HTT01, VY-FXN01, Tau Program, and VY-NAV01. The company was founded by Guang Ping Gao, Mark A. Kay, Krystof Bankiewicz, and Phillip Zamore in June 2013 and is headquartered in Lexington, MA.
相关临床试验
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2014
进行中（未招募）
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33.3%
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- Neurocrine Biosciences has agreed to acquire Soleno Therapeutics, a biopharmaceutical company developing novel therapeutics for rare diseases, in a $2.9 billion transaction. - The acquisition represents Neurocrine's strategic expansion into the rare disease therapeutic space, building on its existing portfolio of specialized treatments. - Soleno focuses on developing and commercializing innovative therapies specifically targeting rare disease populations with significant unmet medical needs. - This marks another major acquisition for Neurocrine following its 2023 purchase of Diurnal, demonstrating continued consolidation in the rare disease sector.
- Vima Therapeutics secured $100 million in funding to develop VIM0423, an oral combination therapy targeting muscarinic cholinergic receptors for movement disorders. - The company plans to initiate mid-stage clinical trials for Parkinson's disease and dystonia, with results expected in 2027. - VIM0423 addresses a significant unmet need, as approximately 160,000 Americans with isolated dystonia currently lack FDA-approved oral treatment options. - The drug is designed to offer improved safety and tolerability compared to existing therapies that target similar receptors.
- Johnson & Johnson terminated its mid-stage AuTonomy study of posdinemab after the anti-tau antibody failed to show statistically significant reduction in cognitive decline compared to placebo in over 500 early-stage Alzheimer's patients. - The failure adds to a string of setbacks for tau-targeting treatments, following previous disappointments from Eli Lilly's LY3372689 and zagotenemab, and Roche's semorinemab programs. - Posdinemab's failure could cast doubt on the therapeutic potential of targeting tau tangles, one of the hallmark features of Alzheimer's disease alongside amyloid plaques. - The setback may impact enthusiasm for similar tau-targeting approaches being developed by Biogen, UCB, Voyager Therapeutics, and other companies in the field.
- Transition Bio and Voyager Therapeutics have entered a drug discovery collaboration to develop novel small molecules targeting TDP-43 pathology in ALS and frontotemporal dementia. - The partnership leverages Transition Bio's biomolecular condensate technology to address TDP-43, which affects over 90% of ALS cases and up to 45% of FTD cases. - Under the agreement, Transition Bio received a single-digit million-dollar upfront payment and is eligible for up to $500 million in milestone payments plus royalties. - The collaboration aims to correct TDP-43 mislocalization while preserving the protein's essential cellular functions, addressing a historically challenging therapeutic target.
- Voyager Therapeutics introduced its Voyager NeuroShuttle platform, a nonviral delivery system that demonstrated sustained brain expression over three weeks in murine studies, significantly longer than transferrin receptor shuttles. - The company entered a collaboration with Transition Bio to develop small molecules targeting TDP-43 for ALS and FTD, addressing a historically undruggable target found in over 90% of ALS cases. - Voyager's tau-targeting programs continue to advance with VY7523 anti-tau antibody in final MAD trial cohort and VY1706 gene therapy expected to enter clinical trials in 2026. - The company maintains a strong cash position of $229 million with runway extending into 2028, while R&D expenses increased to $35.9 million in Q3 2025 to support advancing clinical programs.
- Voyager Therapeutics presented preclinical data on VY-1706, a single-dose AAV gene therapy that delivers tau-silencing siRNA intravenously to treat Alzheimer's disease. - The therapy demonstrated over 50% tau protein reduction in key brain regions including cortex and hippocampus in nonhuman primate studies, with no observed toxicity or clinical signs. - VY-1706 showed robust pharmacologic activity in the P301S mouse model of tauopathy, significantly reducing tau mRNA, protein expression, and pathologic tau accumulation. - The company plans to initiate GLP toxicology studies later this year and aims to submit an investigational new drug application in 2026.
- Voyager Therapeutics has launched a new gene therapy program targeting APOE4, the strongest genetic risk factor for Alzheimer's disease, using its proprietary TRACER capsid technology for intravenous delivery. - The bifunctional therapy simultaneously reduces harmful APOE4 expression while delivering protective APOE2 variant, achieving 90% reduction of APOE4 in key brain regions in preclinical studies. - The company's expanded Alzheimer's franchise now includes four wholly-owned assets targeting tau, amyloid, and APOE pathways, with first human data expected in late 2025. - Despite promising preclinical results, the stock has declined 67% year-to-date as investors weigh execution risks against the potential for breakthrough treatment in a market projected to exceed $50 billion by 2030.
- Vima Therapeutics launched with $60 million Series A funding led by Atlas Venture to advance VIM0423, a potential first-in-class oral therapy for dystonia targeting muscarinic cholinergic receptors. - The company's lead candidate addresses an unmet medical need for over 100,000 Americans with isolated dystonia, as current treatments fail to target the underlying cause of the condition. - VIM0423 is currently in Phase 1 clinical trials with Phase 2 studies planned for the fourth quarter of 2025, representing a novel approach to movement disorder treatment. - The therapy aims to overcome limitations of existing muscarinic receptor drugs by providing improved pharmacology, efficacy, and safety profiles for patients with this disabling neurological condition.
- Voyager Therapeutics published the first peer-reviewed study demonstrating alkaline phosphatase (ALPL) receptor's ability to transport novel AAV capsids across the blood-brain barrier in Molecular Therapy. - The company's VCAP-102 capsid showed 20- to 400-fold increased gene transfer across multiple brain regions compared to AAV9 in both rodents and non-human primates. - Second-generation CNS capsids achieved remarkable transduction rates, including up to 98% of dopaminergic neurons and 94% of motor neurons in non-human primate studies. - Two gene therapy programs utilizing this technology are advancing toward IND filings this year with partners, potentially addressing multiple neurological diseases.
- Voyager Therapeutics announces strategic shift in their SOD1 ALS gene therapy program after three-month non-human primate data indicated the need for alternate payload evaluation. - The company maintains its novel capsid component showing promising activity and tolerability, while postponing the previously planned mid-2025 IND filing for VY9323. - Despite this setback, Voyager's other gene therapy programs remain on track, including partnerships with Neurocrine Biosciences for GBA1 Parkinson's disease and Friedreich's ataxia initiatives.
