India, Aug. 31 -- image credit- shutterstock

Cell and gene therapy, long hailed as one of the most promising frontiers in modern medicine, has offered hope of life-changing treatments for patients with rare and serious diseases.

The safety concerns began in March 2025, when Sarepta Therapeutics reported the first patient death linked to its approved Duchenne muscular dystrophy gene therapy, Elevidys. The non-ambulatory boy developed acute liver failure after receiving the treatment. In June 2025, a second non-ambulatory patient died following Elevidys treatment, prompting Sarepta to introduce a series of safety measures, including adding a black box warning to the product label.

In May 2025, Rocket Pharmaceuticals disclosed the death o...