The next chapter in the XLMTM gene therapy story

There’s a new twist in the story of a gene therapy for X-linked myotubular myopathy from Astellas that led to four patient deaths in 2021 and precipitated global concerns about the safety of AAV vectors: it works, well. 

The revelation that the gene therapy appears to be changing the course of the severe musculoskeletal disease, with the majority of patients coming off ventilators and gaining motor functions, raises important questions about how to improve the safety of AAV vectors, and how much risk regulators should tolerate for therapies that can modify the most devastating diseases...