Two patients die in Audentes gene therapy study, heightening concerns over high-dose treatments

Two children with a rare neuromuscular disease have died after receiving a high dose of a gene therapy in a clinical trial run by Audentes Therapeutics, according to two letters sent by the company to patient groups. Both of the children suffered liver problems that ultimately led to sepsis. The most recent death occurred this month, though the letters revealed Audentes halted dosing of new patients before either occurred. The Food and Drug Administration subsequently placed the study on a formal clinical hold. The gene therapy, called AT132, is designed to treat X-linked myotubular myopathy, a deadly disease caused by mutations in a single gene. Audentes, which was bought by Japan’s Astellas Pharma for $3 billion in December, aimed to submit the treatment for approval this year, but those plans are now on hold, according to the letter.

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