2 patients die in Audentes gene therapy study – Becker’s Hospital Review


Alia Paavola –  | 

Two patients in Audentes Therapeutics’ study testing a gene therapy for a rare muscle disorder called X-linked myotubular myopathy have died, according to STAT. 

The San Francisco-based drugmaker said in a letter June 23 that the patients died after experiencing liver problems that appeared to be linked to its experimental drug. 

X-linked myotubular myopathy is a condition that causes severe muscle weakness, and most patients with the disorder don’t survive childhood.

Audentes CEO Natalie Holles told STAT that the company has stopped enrolling new patients in the study, and shortly after the company’s decision, the FDA placed a clinical hold on the study. 

Ms. Holles said that the company will investigate the two deaths thoroughly. 

“While this is a terrible loss and it’s heartbreaking, we see a path forward here, and we’re redoubling our efforts as a company to get products to patients as quickly as possible and as broadly as possible, recognizing that we have to do that safely,” Ms. Holles told STAT.

Read the full report here

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