Voyager Therapeutics Presents Data for Second-Generation, TRACER-Generated Capsids and CNS Gene Therapy Programs Advancing Toward Clinical Trials at the ASGCT 27th Annual Meeting

- Translational potential of TRACER-derived AAV capsids demonstrated by data across multiple species (murine, porcine, multiple NHPs, human cells) and receptor binding -

- Preclinical safety and efficacy demonstrated in Alzheimer’s disease and ALS gene therapies; low IV doses provided broad CNS biodistribution and robust transduction of key cell types -

LEXINGTON, Mass., May 08, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced the presentation of data related to its TRACER capsid discovery platform and TRACER-driven gene therapy programs at the American Society of Gene & Cell Therapy’s (ASGCT) 27th annual meeting.

Second-generation, intravenously (IV)-delivered capsids, evolved through the TRACER platform, showed further enhanced blood-brain barrier (BBB) penetrance, greater liver detargeting, and transduction of 50-75% of cells across diverse brain regions, with upwards of 95% transduction in certain key cell types such as Purkinje Neurons. Further, a gene therapy in preclinical development 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 non-human primate (NHP) spinal cord motor neurons following a single IV delivery at a clinically relevant dose of 3E13 vg/kg. The potential translatability of these capsids is supported by data across multiple species, including mice and multiple species of NHP, as well as binding to Alkaline Phosphatase (ALPL, formerly called Receptor X) in multiple species and in human cells. These data are highlighted in the oral presentation, “Continued directed evolution of VCAP-101 and VCAP-102 identifies second generation capsids with increased brain tropism in non-human primates and mice (#119)” on Wednesday, May 8, 2024, 4:30 p.m. – 4:45 p.m. ET, as well as in multiple posters detailed below.

“Results across species, capsid generations, and disease models provide the most extensive validation to date of the high translational potential of our TRACER capsids for gene therapy in the CNS,” said Todd Carter, Ph.D., Chief Scientific Officer of Voyager Therapeutics. “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 IND filings in 2025.”