Moving Forward after Two Deaths in a Gene Therapy Trial of Myotubular Myopathy
Moving Forward after Two Deaths in a Gene Therapy Trial of Myotubular Myopathy 1 The sponsor of this clinical study, Audentes Therapeutics (which was acquired by Japan’s Astellas Pharma in 2019), had generated impressive efficacy studies in a canine model of MTM 2 and conducted non-primate (NHP) safety studies before advancing into the clinic. 3 The initial cohort of six pediatric MTM patients dosed at 1×10 14 genome copies (GC)/kg also showed encouraging results. However, all three patients administered a three-fold higher dose (i.e. 3×10 14 GC/kg) experienced severe hepatotoxicity, wh...
Source: genengnews.com