Voyager Therapeutics

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Founded: 2014

Location: Cambridge, MA

Employee range: 200 - 500

Clinical stage: RD

Therapy area: Alzheimer's

Drug types: NEU, RAR

Lead product: Undisclosed

Funding: $60M B Apr 2015

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Business:

AAV capsid Gene Therapy

Drug notes:

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

About:

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.