Astellas Reports Third Patient Death in Audentes Gene Therapy Study
Summary
San Francisco-based Audentes, a subsidiary of Japanese pharmaceutical company Astellas Pharma, said Friday that early findings indicate that gastrointestinal bleeding was the cause of the most recent death. The Audentes gene therapy, dubbed AT132, is in development as a treatment for X-linked myotubular myopathy, an inherited disorder that causes extreme muscle weakness, respiratory failure, and early death. The patient who most recently died began showing signs of liver dysfunction within three to four weeks after receiving the gene therapy, Audentes says. In 2018, the FDA halted a Solid Biosciences (NASDAQ: SLDB) study evaluating its AAV-based gene therapy for Duchenne muscular dystrophy after complications led to the hospitalization of one patient. Though the FDA approved Zolgensma last year for children younger than 2, Novartis has been conducting additional tests to support use of the AAV-based gene therapy in treating older SMA patients.
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