Individualized Therapeutics Could Get Boost From NIH Platform Gene Therapy Pilot
Experience and data from the study, which will use the same vector platform to deliver different transgenes for different diseases, could feed into the US FDA’s efforts to establish a public-private partnership to enable manufacturing of gene therapies for ultra-rare diseases.
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Advisory committee will discuss five types of toxicity risks with adeno-associated virus vector-based gene therapy products at a two-day meeting in September, which follows some recent high-profile instances of adverse events that have delayed development programs.
US FDA’s Peter Marks says companies are not exchanging information about adenoviral factors, which is probably slowing down the field. He describes NIH's plans for a bespoke gene therapy consortium for vector generation and continued efforts for regulatory harmonization.