FDA Regulation On Gene Therapy | The Brainstorm EP 96

FDA Regulation and Drug Approval - The FDA's drug and biological approvals, including gene therapies, are under scrutiny following the departure of Dr Assad [3] - The FDA commissioner aims to recalibrate standards for more efficient regulatory pathways, leveraging AI and big data to improve the drug approval process [10][11] - The industry anticipates the incoming head of the division handling biologics to share the same priorities of improving the FDA and facilitating innovation [12] - The FDA is considering approving rare disease therapies at the first sign of promise, recognizing the lack of meaningful disease-modifying options for these patients [19] Gene Therapy and Clinical Trials - A gene therapy from Sarepta Therapeutics for Duchenne muscular dystrophy faced safety concerns due to patient deaths, leading to a temporary halt of shipments [4][5] - Patient advocacy groups expressed devastation over losing the gene therapy option, which could potentially halt or reverse disease progression [6] - Acute liver failure was linked to the deaths of teenage boys in the Sarepta Therapeutics trial, potentially due to higher doses per kilogram in older patients [13][14] - AI can be embedded into clinical trials to simulate and explore avenues, potentially preventing patient deaths and improving the success rate of getting the right drugs to the right patients faster [16][17] Genomics and AI - The industry is excited about the potential of gene editing for common diseases like cardiovascular disease, building on the proof of concept in rare diseases [21] - CRISPR Therapeutics is developing gene editing therapies targeting genes involved in liver metabolism to address cardiovascular disease [22] - Advances in AI are unlocking new possibilities in genomics, enabling better target design, faster pre-clinical studies, and improved clinical trial design [30][31][32]