Astellas is retrying its gene therapy for X-linked myotubular myopathy (XLMTM) after a prior trial was paused due to patient deaths. The company is resuming clinical development with a redesigned approach, according to a report from STAT News. This marks a significant attempt to revive a therapy for a rare and often fatal neuromuscular disease.
The earlier trial had been halted after several patients died, raising serious safety concerns about the high-dose gene therapy. XLMTM primarily affects newborn boys, causing severe muscle weakness and respiratory failure. Astellas acquired the therapy through its purchase of Audentes Therapeutics in 2020 for $3 billion.
The new trial will use a lower dose of the gene therapy vector, aiming to reduce toxicity while maintaining efficacy. STAT reported that the company has received regulatory clearance to proceed with the revised protocol. Specific enrollment numbers and trial sites have not yet been disclosed.
If successful, the therapy could become a life-changing treatment for a disease with no approved options. However, the shadow of past fatalities may complicate patient recruitment and regulatory oversight. Astellas faces the challenge of restoring confidence among clinicians and families.
Critics argue that gene therapy risks remain poorly understood, especially in vulnerable populations. Some patient advocates have called for more transparency in trial design and safety monitoring.