Our Mission at Voyager Therapeutics: Define the Future of Neurogenetic Medicines

We are in an unprecedented time in neurotherapeutics. Medicines that address the causative disease biology underlying central nervous system (CNS) diseases such as Alzheimer’s disease and amyotrophic lateral sclerosis (ALS) are becoming a reality for patients.

Despite these advancements, progress is limited by delivery challenges, as the blood-brain barrier prevents the uptake of many investigational therapies. At Voyager Therapeutics, we are committed to overcoming this challenge and delivering transformative neurogenetic medicines to patients in need. We are approaching this with our TRACER™ (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform. Voyager has leveraged TRACER to create novel capsids that harness the extensive vasculature of the CNS to cross the blood-brain barrier, with the potential to enable gene therapies for CNS diseases.

TRACER-generated capsids have been shown in preclinical studies to transduce a broad range of CNS regions and cell types following intravenous delivery, while simultaneously de-targeting the liver, an organ often implicated in side effects. In cross-species preclinical studies (rodents and multiple non-human primate species), intravenous delivery of TRACER-generated capsids resulted in widespread payload expression across the CNS at relatively low doses. These studies enabled selection of multiple development candidates in Voyager’s wholly-owned and partnered gene therapy programs for neurologic diseases, which are now advancing towards human clinical trials.

The strong performance of our capsids has enabled selection of three development candidates in Voyager’s wholly-owned and partnered gene therapy programs for neurologic diseases, which are currently advancing towards anticipated investigational new drug (IND) application filings in 2025.

Voyager’s team presented data for our second-generation, TRACER-generated capsids and CNS gene therapy programs advancing toward clinical trials.

Through our TRACER™ platform, we are reaching CNS targets once thought unreachable. Key findings presented at this year’s ASGCT annual meeting across species, capsid generations, and disease models provided the most extensive validation to date of the high translational potential of our TRACER capsids for gene therapy in the CNS:

• Second-generation intravenously (IV)-delivered capsids, evolved through the TRACER platform, showed further enhanced BBB penetrance, greater liver detargeting, and transduction of 50-75% of cells across diverse brain regions.
• Further, a gene therapy in preclinical development in our pipeline for SOD1 amyotrophic lateral sclerosis (ALS), which combines a SOD1 RNAi transgene packaged in a second-generation capsid, reduced SOD1 messenger RNA (mRNA) expression by up to 80% in NHP spinal cord motor neurons following a single IV delivery at a dose of 3E13 vg/kg.
• The potential translatability of our capsids is supported by data across multiple species, including mice and multiple species of non-human primates, as well as binding to Alkaline Phosphatase (ALPL, formerly called Receptor X) in multiple species and in human cells.

Voyager’s novel TRACER-derived capsids underlie 13 partnered programs and three wholly-owned programs to enable IV-delivery of gene therapies for diseases of the central nervous system. We are proud of the progress of our programs, propelled by our vision to help create a world in which transformative treatments and cures are available to the millions afflicted with neurological diseases.