Despite impressive gains in recent years, investment in gene therapy products is at a critical juncture due to current manufacturing challenges, among other factors, says Peter Marks, director of CBER, speaking at the 2023 PDA/FDA Joint Regulatory Conference on Tuesday.

Gene therapy is “finally really coming of age,” Marks pronounced, highlighting that the FDA has now approved 15 gene therapies including stem cell, chimeric antigen receptor (CAR)-T cell and directly administered products, with more therapies “coming along in the coming months and years, so this is an exciting time for gene therapy particularly as it applies to very small populations.”

But, Marks warned, there are some “real challenges” in gene therapy development right now, noting that “small batch gene therapy is something that is becoming really challenging because of the manufacturing costs,” with clinical development timelines meaning it will take companies approximately “five years to get that money back out” from initial investment.

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