The clinical trial had already been placed on hold in connection with the two prior patient deaths, which were linked to the higher dose of 300 trillion viral vectors per kilogram, while no deaths have been reported among those receiving the lower dose of 100 trillion vectors.
The company disclosed in a letter to the X-linked myotubular myopathy patient community that a second child in its Phase I/II study had died. A formal clinical hold has been placed on the trial.
CEO and neuroscientist Dr. Iftach Dolev on how the new device can bring a powerful tool to psychiatrists and neurologists alike.
Shares of Audentes rose more than 100% following news that the Japanese drugmaker would acquire it. The company's lead candidate is a gene therapy for a rare muscular weakness disorder, currently in a Phase I/II study for which it presented data in October.