In 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.
Shares of gene therapy stocks fell. Avexis’ (AVXS) stock dropped 8 percent initially but has since rebounded. Solid Biosciences (SLDB), which went public just last week, fell 17 percent. Sarepta Therapeutics (SRPT) was down 2 percent.
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