Next-generation AAV capsids hold key to developing life-changing therapies

May 24, 2022

Voyager Therapeutics’ discovery platform is identifying novel capsids targeting desired cells and tissues with greater specificity, at lower doses, and with fewer off-target risks than conventional adeno-associated virus (AAV) serotypes in non-human primates (NHPs), contends Todd Carter, PhD, senior vice president for research.

The company's Tropism Redirection of AAV by Cell-type-specific Expression of RNA (TRACER) platform has demonstrated superior and widespread gene expression in the central nervous system compared to conventional AAV capsids, as well as cell- and tissue-specific transduction including to areas of the brain that have been traditionally difficult to reach, according to Carter.

"We use transduction itself. What that means is we're not just looking at the genome of the vector going into the various tissues, the brain, etc., but we're looking for expression off of that genome. Using that RNA-level of expression really makes the difference in determining not only the delivery of the viral genome but also the functional delivery of that viral genome," Carter told ScienceBoard.net at the American Society of Gene & Cell Therapy (ASGCT) 2022 annual meeting in Washington, DC.

Voyager Therapeutics claims multiple TRACER capsids have achieved broad transgene expression with a top variant achieving more than 1,000-fold increased expression versus conventional AAV9 across several brain regions in NHPs.

Watch the video below to learn more.


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Last Updated 5/24/2022 2:08:10 PM