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.
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.
The company, which is also working on 18 other therapeutics, said in its statement that the teams’ work is essential not only to shed light on the study’s outcome but also “on the challenges of gene therapy broadly.” Meanwhile, it said, “we will continue to work with our researchers, collaborators, and partners to develop therapies for the neuromuscular diseases in our pipeline.”
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