n a newly published paper, a well-regarded gene therapy pioneer has raised concerns about severe neurological and liver toxicity observed in animals treated with high doses of a gene therapy targeted against a rare and fatal neuromuscular disorder.

The scientist, Dr. James Wilson, had previously quit the board of Solid Biosciences (SLDB), over concerns that the virus the company uses in its gene therapy, being developed for patients with a form of muscular dystrophy, was too dangerous. In Tuesday’s paper, however, he and others laid out those concerns in more detail.

This is a STAT Plus article and you can unlock it by subscribing to STAT Plus today. It's easy! Your first 30 days are free and if you don't enjoy your subscription you can cancel any time.
Already a subscriber? Log in here.

Leave a Comment

Please enter your name.
Please enter a comment.

Sign up for our Morning Rounds newsletter

Your daily dose of what’s new in health and medicine.