Voyager Therapeutics Inc
NASDAQ:VYGR
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Voyager Therapeutics Inc
Voyager Therapeutics, Inc. is a clinical-stage gene therapy company, which engages in the development of treatments for severe neurological diseases. The company is headquartered in Cambridge, Massachusetts and currently employs 101 full-time employees. The company went IPO on 2015-11-11. Its gene therapy platforms enable it to engineer, optimize, manufacture, and deliver its adeno-associated virus (AAV)-based gene therapies. The company is identifying AAV capsids, the outer viral protein shells that enclose genetic material of a virus payload. The company has developed a AAV capsid discovery platform, Tropism Redirection of AAV by Cell Type-Specific Expression of RNA (TRACER) to facilitate the selection of AAV capsids with blood brain barrier (BBB) crossing and cell-specific transduction properties for therapeutic applications. Its gene therapy programs include treatment programs for Huntington's disease, monogenic amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), and various diseases linked to GBA1 mutations, including Parkinson's disease, Lewy body dementia and Gaucher's disease. The company also focuses on tauopathies and indications in neuro-oncology.
Voyager Therapeutics, Inc. is a clinical-stage gene therapy company, which engages in the development of treatments for severe neurological diseases. The company is headquartered in Cambridge, Massachusetts and currently employs 101 full-time employees. The company went IPO on 2015-11-11. Its gene therapy platforms enable it to engineer, optimize, manufacture, and deliver its adeno-associated virus (AAV)-based gene therapies. The company is identifying AAV capsids, the outer viral protein shells that enclose genetic material of a virus payload. The company has developed a AAV capsid discovery platform, Tropism Redirection of AAV by Cell Type-Specific Expression of RNA (TRACER) to facilitate the selection of AAV capsids with blood brain barrier (BBB) crossing and cell-specific transduction properties for therapeutic applications. Its gene therapy programs include treatment programs for Huntington's disease, monogenic amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), and various diseases linked to GBA1 mutations, including Parkinson's disease, Lewy body dementia and Gaucher's disease. The company also focuses on tauopathies and indications in neuro-oncology.
Pipeline Progress: Voyager advanced VY-1706, its tau silencing gene therapy, into IND-enabling studies with plans for IND filing in 2026. The SOD1 program moved back to research after payload setbacks.
Clinical Data: VY-1706 showed promising tau mRNA knockdown (50% to 73%) broadly across the brain in non-human primate studies. VY-7523 anti-tau antibody displayed dose-proportional PK, no serious adverse events, and CSF to serum ratio similar to approved antibodies.
Financial Strength: Voyager ended 2024 with $332 million in cash and has $8.2 billion in potential future milestone payments from partnerships. Cash runway is guided to mid-2027, excluding upside from future milestones.
Partnership Milestones: Two Neurocrine-partnered gene therapy programs are expected to enter the clinic this year, with significant near-term developmental milestones possible.
Strategic Focus: The company remains committed to partnerships for non-dilutive funding and is open to additional business development. Management is also excited about ongoing work with ALPL nonviral shuttles for CNS delivery.
Upcoming Catalysts: Data will be presented at major conferences (AD/PD in April, AAIC in July, CTAD in the fall), with anticipated third-party readouts in the tau space that could impact Voyager’s programs.
