Voyager Therapeutics

AAV capsid Gene Therapy

13+ additional undisclosed programs RD ALS, Friedreich's ataxia, Parkinson's, rare neurological disease, CNS diseases, undisclosed

Voyager Therapeutics is advancing delivery modalities for gene therapies. Many gene therapies are in development, yet delivery of the therapy is still a major limitation. AAVs are a popular choice but require high doses and have off-target effects. Voyager is using its TRACER screening platform to enhance AAV capsids’ specificity and ability to target desired cells and tissues. Voyager’s AAVs also pass the blood-brain barrier, offering the potential to cure neurological diseases. Initial non-human primate data demonstrate the selectivity of TRACER-derived capsids to target the CNS and cardiac muscle. These capsids may enable single-dose treatment for a broad spectrum of diseases.