How This Sleeper Biotech, With A 687% Gain, Is Making A Comeback
Is shrugging off several messy years in gene therapy development, an analyst said Thursday as he initiated coverage of the biotech stock with an outperform…
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Is shrugging off several messy years in gene therapy development, an analyst said Thursday as he initiated coverage of the biotech stock with an outperform…
Pfizer has a pipeline with more than 90 drugs in development. Pfizer is developing two drugs capable of generating more than a billion sales. Competition is fierce but Pfizer is in the race.
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...