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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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.
The company, which has treatments in the clinic for CLN2 Batten disease and APOE4-associated Alzheimer’s disease, is developing AAV-mediated gene therapies designed for safer outcomes.
Lack of reference standards inhibits ability to make comparisons between products or set caps for total vector genome dose or total capsid dose, advisory committee says; panelists suggest other approaches to prevent and mitigate toxicities, including running longer-term animal studies and investigating the efficacy of immunosuppression prophylaxis and treatment strategies.