At a regulatory forum hosted by the Food and Drug Administration on Thursday, gene therapy experts and advocates conveyed serious concerns about the sector’s challenges, despite its remarkable scientific achievements.
The meeting occurred in the wake of a notable success story – 10-month-old KJ Muldoon’s return home from a Philadelphia hospital after receiving a customized CRISPR treatment for his rare liver condition, CSP1 deficiency. While not completely cured, the treatment has enabled him to resume normal eating and potentially avoid a liver transplant.
However, the 23 experts who gathered at the FDA’s White Oak, Maryland headquarters painted a sobering picture of an industry in crisis. Despite having dozens of approved cell and gene therapies in the United States offering near-curative potential for serious conditions like sickle cell disease and acute lymphoblastic leukemia, the sector faces significant hurdles.
According to Terence Flotte, dean of the University of Massachusetts’ T.H. Chan School of Medicine, more than 100 rare disease gene therapy products that had reached clinical trials have been discontinued since 2023, not due to treatment failure but because of anticipated market challenges. Investment in genetic medicine has declined as companies struggle with profitability concerns, leading to research cuts, staff reductions, and some business closures.
Stanford University professor Crystal Mackall emphasized the disconnect between scientific progress and practical implementation, noting that despite “spectacular” advances, the field struggles to deliver treatments to all potential beneficiaries. Large
pharmaceutical companies have become increasingly hesitant to invest billions in overcoming regulatory and reimbursement obstacles.
FDA leadership, including Commissioner Martin Makary and cell and gene therapy office head Vinay Prasad, showed understanding of these concerns. Makary pledged to improve regulatory efficiencies while maintaining the FDA’s facilitative approach. Prasad expressed support for flexible trial designs when appropriate and acknowledged that progress often comes incrementally rather than in breakthrough leaps.
Experts proposed various solutions during the meeting. Carl June, an immunologist from the University of Pennsylvania, suggested adopting elements of China’s two-tier regulatory system, which enables faster initiation of first-in-human trials under local review board supervision. UCLA scientist Don Kohn advocated for reduced
“comparability” testing requirements when transitioning from academic to commercial production.
The importance of regulatory incentives, such as priority review vouchers, was highlighted as crucial for capital generation. Many participants emphasized the need for increased sharing of feedback and lessons learned from industry applications.
A recurring theme throughout the discussion was the risk of losing U.S. leadership in developing curative treatments if regulatory challenges aren’t addressed. As June warned, “If we don’t adapt, the next generation of treatments will emerge abroad.”
The message appeared to resonate with FDA leadership. Prasad indicated plans to make more internal documents available to help developers understand the agency’s requirements. Makary emphasized the meeting’s purpose as “an honest listening session” rather than a ceremonial exercise.
The context of these discussions was highlighted by CRISPR scientist David Liu’s observation that approximately 10 million babies are born annually with one of about 10,000 known rare genetic diseases, many of which could potentially be treated with genetic medicines. This underscores the critical importance of addressing the sector’s challenges to realize the potential of providing customized genetic treatments at scale.
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