This segment focuses on the research, development, and clinical application of gene therapy technologies to treat a range of neurological disorders. Voyager Therapeutics employs adeno-associated virus (AAV) vectors to deliver therapeutic genes directly to the brain, targeting the underlying genetic causes of diseases like Parkinson's, Alzheimer's, Huntington's, and ALS. R&D activities include optimizing AAV capsid design for improved brain penetration and gene expression, as well as conducting preclinical and clinical studies to evaluate the safety and efficacy of gene therapy candidates. The goal is to develop disease-modifying therapies that can halt or reverse the progression of these debilitating conditions, offering hope for patients with limited treatment options. Partnerships with companies like Neurocrine Biosciences and AbbVie support the development and commercialization of these innovative gene therapies.

Voyager Therapeutics' TRACER™ (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) platform is a groundbreaking technology for discovering novel AAV capsids with enhanced tropism and delivery capabilities to specific cell types within the central nervous system. This platform enables the development of gene therapies with improved targeting and reduced off-target effects. R&D efforts are focused on generating and screening vast libraries of AAV capsids to identify those with optimal characteristics for delivering therapeutic genes to specific brain regions affected by neurological diseases. The TRACER™ platform has the potential to revolutionize gene therapy by enabling the development of more effective and safer treatments for a wide range of neurological disorders. Licensing agreements for next-generation capsids further expand the reach and impact of this innovative technology.

Voyager Therapeutics is dedicated to advancing a robust pipeline of neurogenetic medicines targeting a spectrum of severe neurological diseases. This includes programs focused on Alzheimer's disease (VY-TAU01, APOE4 gene therapy), amyotrophic lateral sclerosis (VY-SOD102), Huntington's disease (VY-HTT01), and Friedreich's ataxia (VY-FXN01). Research and development efforts encompass preclinical studies, clinical trials, and strategic collaborations to accelerate the development of these therapies. The pipeline leverages gene therapy and other therapeutic approaches to address the underlying genetic causes of these diseases, with the goal of providing transformative treatments that can improve the lives of patients and their families. Voyager's commitment to neurogenetic medicine is reflected in its ongoing efforts to expand its pipeline and advance innovative therapies through the clinic.