Gene Therapies: US Panel To Weigh Strategies For Preventing, Mitigating AAV Vector Toxicities
FDA seeks advice on screening patients potentially at higher risk for liver and other injuries, strategies to implement before or after gene therapy administration to head off or mitigate potential adverse events, and whether upper limits should be set for the total vector genome dose and total capsid dose.
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BioMarin’s PKU Gene Therapy Clinical Hold To Last ‘Several Quarters’
BMN 307 for phenylketonuria was put on a clinical hold in September due to liver cancer observed in mice and was expected to lift in Q1 of 2022, but the US FDA has requested additional preclinical studies.
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.
Gene Therapy Situation Gets More Awkward With Another Death In Astellas XLMTM Study
An expert pointed out that XLMTM patients are already at high risk, but if linked to therapy toxicity, the death could doom the ASPIRO trial.