The death occurred less than two weeks after Astellas Pharma reported that the patient developed liver problems after being dosed with the experimental gene therapy for a rare neuromuscular disorder. Astellas said the cause of death is still under investigation, and the FDA has placed a clinical hold on the study.
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.
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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.
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.
The Alliance for Regenerative Medicine and 13 biotech companies released a statement outlining bioethical principles on genome editing, including opposition to modifications that can be passed to future generations.
An analyst wrote that the therapy's combination of AAV delivery with a validated means of treating DMD could distinguish it from other gene therapies in what is already a crowded space.
The home care industry is booming. As Baby Boomers age and more families seek in-home solutions for loved ones, demand for home care services is at an all-time high.
Hospitals - especially in Europe - anticipate growing interest from international patients. But a wave of industry consolidation is debatable.
French firm to start trial of Dyn101, in-licensed from US-based Ionis Pharmaceuticals, in centronuclear myopathy next year.
Audentes Therapeutics, a San Francisco-based gene therapy startup, just raised $65 million to advance three candidates to the clinic that treat X-linked myotubular myopathy, Pompe disease and a rare form of tachycardia called CPVT.