Two patients die in now-halted study of Audentes gene therapy
By Ben Fidler,
BioPharma Dive
| 06. 26. 2020
Dive Brief:
- 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.
Dive Insight:
Gene therapy has emerged rapidly in recent years because the field overcame safety concerns — most notably, the tragic death of teenager Jesse Gelsinger in a clinical trial in 1999 — that cooled initial optimism and slowed research...
Related Articles
The Center for Genetics and Society is delighted to recommend the current edition of GMWatch Review – Number 589. UK-based GMWatch, a long-standing ally, was founded in 1998 by Jonathan Matthews as an independent organization seeking to counter the enormous corporate political power and propaganda of the GMO industry and its supporters. Matthews and Claire Robinson are its directors and managing editors.
CGS works to ensure that social justice, equity, human rights, and democratic governance are front...
By Ryan Cross, Endpoints News | 08.19.2025
Human eggs are incredibly rare cells. The ovary typically produces only 400 mature eggs across a woman’s life. But biologists in George Church’s lab at Harvard University — a group that’s never content with nature’s limits — just got a...
By Katherine Drabiak, Journal of Medical Ethics Forum | 08.07.2025
Adapted from Mitochondrial DNA at
National Human Genome Research Institute
Recently, media outlets around the world have been reporting on children born from pronuclear genome transfer (sometimes called “3-parent IVF,” “mitochondrial donation” or “mitochondrial replacement therapy”) at Newcastle Fertility Center...
By Nicky Hudson, The Conversation | 08.12.2025