The Japanese drugmaker is paying $18 million upfront to exercise an option on the AAV platform – which my help avoid safety issues that have emerged with other AAV-based gene therapies – with milestone and royalty payments that could exceed $1.6 billion.
Those safety issues have hit Astellas’ aspirations in gene therapy directly. In September, it reported a fourth patient death in a clinical trial of its AT132 (resamirigene bilparvovec) gene therapy for the rare disease X-linked myotubular myopathy (XLMTM), possibly linked to liver side effects.
