After gene therapy deaths, Astellas tries potentially safer approach

By Jason Mast June 8, 2023 Astellas Pharma said Thursday it will license and

By Jason Mast June 8, 2023

Astellas Pharma said Thursday it will license and develop a new gene therapy for a devastating muscle disorder, after four boys died in a clinical trial testing an earlier treatment.

The hope is that the new therapy will allow researchers to treat the disease, known as X-linked myotubular myopathy, or XLMTM, with much lower doses of the viruses used to shuttle genes into patients’ cells. In theory that should minimize the risk of severe side effects.

"The lower the dose we give, the lower the risk is going to be," said Alan Beggs, the Boston's Children's Hospital researcher who helped develop the earlier treatment and is a scientific co-founder of the startup behind the new drug.

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General Assignment Reporter

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients.

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gene therapy

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After gene therapy deaths, Astellas tries potentially safer approach