Deaths associated with Astellas Pharma’s gene therapy program for patients with X-linked Myotubular Myopathy underscore some of the risks of developing potential one-and-done treatment options for serious diseases.


A patient treated with Astellas Pharma’s experimental gene therapy for patients with X-linked Myotubular Myopathy (XLMTM) died after reports of a serious adverse event following treatment with AT132.


Astellas Pharma paused the screening and dosing of an investigational gene therapy for patients with X-linked Myotubular My-opathy (XLMTM) following a serious adverse event report in a trial participant.

The U.S. Food and Drug Administrationlifted a clinical hold on Audentes Therapeutics’ ASPIRO trial studying AT132 as a potential treatment for X-linked myotubular myopathy (XLMTM), a rare neuromuscular disease.

San Francisco-based Audentes Therapeutics reported two deaths in the company’s gene therapy clinical trial for AT132 for X-linked myotubular myopathy.

Tokyo-based Astellas Pharma announced plans to acquire San Francisco-based Audentes Therapeutics for $60 per share in cash, representing a total equity value of about $3 billion.