The lone volunteer in a unique study involving a gene-editing technique has died, and those behind the trial are now trying to figure out what killed him.
Terry Horgan, a 27-year-old who had Duchenne muscular dystrophy, died last month, according to Cure Rare Disease, a Connecticut-based nonprofit founded by his brother, Rich, to try and save him from the fatal condition.
At this point, it’s unclear whether Horgan received the treatment and whether CRISPR, other aspects of the study or the disease itself contributed to his death. Deaths are not unheard of in clinical trials, which test experimental treatments and sometimes involve very sick people. Linda Horgan of Montour Falls, N.Y., holds a photo of her three brothers, all of whom died of Duchenne muscular dystrophy. Horgan’s youngest son, Terry, was the lone participant in a unique trial to treat his particular form of Duchenne muscular dystrophy. said multiple teams across the country are looking into the details of the trial and its outcome, and the company intends to share findings with the scientific community.
Bauman said the company has filed a report on death the with the FDA as required. The FDA declined to release or confirm the report. Safety issues have arisen in gene therapy studies before. Late last year, Pfizer reported the death of a patient in its early-stage trial for a different Duchenne muscular dystrophy gene therapy. And in a major earlier setback for the gene therapy field, 18-year-old Jesse Gelsinger died in 1999 during a study that involved placing healthy genes into his liver to combat a rare metabolic disease. Scientists later learned that his immune system overreacted to the virus used to deliver the therapy.
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