Voyager advances gene therapy platform for CNS diseases

By Kate Madden Yee, The Science Advisory Board contributing writer

August 9, 2021 -- Voyager Therapeutics has launched new gene therapy programs for Huntington's disease, amyotrophic lateral sclerosis caused by mutation in the SOD1 gene, spinal muscular atrophy, and diseases linked to glucosylceramidase beta mutations, powered by its proprietary adeno-associated virus (AAV) capsids.

The company's proprietary AAV capsids have effective transgene expression in the brain. The vectors demonstrated robust delivery across the blood-brain barrier and widespread central nervous system transduction in non-human primates compared to AAV9 delivery, the firm said.

Voyager has received more investment in its RNA-driven TRACER (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) AAV screening technology, it announced. Its TRACER program is intended to identify new capsids that overcome existing AAV capsids' limitations. The company is screening nine additional AAV capsid campaigns to further expand its capsid library and identify capsids optimized for specific applications.

In other Voyager news, the company appointed Michael Higgins as its interim chief executive officer and Dr. Glenn Pierce, PhD, as its interim chief scientific officer.

Finally, the company reported second quarter 2021 revenue from collaborations totaling $1.4 million compared to $28.7 million in 2020, a decrease it attributed to reduced revenue due to research services and cost reimbursements with Neurocrine and AbbVie. It also posted a net loss of $30.1 million for the quarter, compared to $8.7 million for the same period last year.

Neurocrine, Voyager tout gene therapy clinical results for Parkinson's disease
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