Gene therapy programs face pivotal moments as safety concerns arise. Sarepta’s multiple deaths linked to its Duchenne and limb-girdle muscular dystrophy gene therapies have drawn FDA action and sparked industry-wide discussions on the risks of AAV-based vectors. Meanwhile, Voyager Therapeutics expanded its Alzheimer’s pipeline with APOE-modulating gene therapy using a blood-brain barrier-penetrant vector. Genetic Leap received FDA clearance for its RNA-based IL-2 modulator, GL-IL2-138, marking progress in immune modulation therapies. Such developments illustrate both the promise and the safety challenges of gene and RNA therapies in the current biotech ecosystem.