AAV Gene Therapy Panel Shined Light On Safety Issues That Sponsors Have Downplayed – FDA’s Bryan
September advisory committee was less about getting advice and more about calling public attention to AAV gene therapy safety issues, OTAT director Wilson Bryan says. FDA is not ready to set a cap on vector dose but could consider doing so down the road.
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Deal Snapshot: Roche and University of Pittsburgh spinout Avista Therapeutics signed the pact to enable the Swiss drug maker to develop gene therapies for retinal diseases using Avista’s platform tech.
Sanofi/Sobi’s efanesoctocog is in line for US FDA action three months after CSL/uniQure’s gene therapy user fee goal, while BioMarin’s Roctavian resubmission and Novo Nordisk’s concizumab are waiting in the wings.
The Swiss giant has gained access to next-generation AAV capsids that could reach cells in the brain, something that has not been possible with technologies used to date.