Two boys have died after receiving high doses of a gene therapy treatment for their rare muscle disease, Biopharma Dive reports. The patients, born with x-linked myotubular myopathy, developed liver problems that apparently led to sepsis, according to a 23 June letter to patient groups from trial sponsor Audentes Therapeutics. They were older patients and had existing liver disease; several younger patients who got lower doses of the treatment have done well and now breathe on their own without a ventilator. The U.S. Food and Drug Administration (FDA) has put the trial on hold. Audentes, which had stopped enrollment before the deaths, has postponed plans to seek FDA approval for the drug this year. Since the 1999 death of gene therapy patient Jesse Gelsinger slowed research, the field has rebounded and FDA has approved two rare disease treatments. But recently, animal studies have suggested high doses of gene therapy can cause dangerous liver toxicity.
sciencemag.org, 29 June 2020
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