1 Abstract The devastating Ebola outbreak in West Africa in 2013-2016 accelerated the progress of several medical countermeasures (MCMs) against Ebola virus disease (EVD). Several IPs were used during that outbreak, but no conclusion on efficacy could be drawn. Only the Randomized Controlled Trial (RCT) on ZMapp was promising but inconclusive. More recently, during the second largest Ebola outbreak in North Kivu and Ituri provinces, Democratic Republic of the Congo (DRC), four IPs (Remdesivir, Regeneron, ZMapp and MAb114) have been evaluated in a RCT, the Pamoja Tulinde Maisha (PALM) study. Two products (Regeneron and MAb114) demonstrated efficacy as compared to the control arm, ZMapp. Very few side effects have been reported. Results from this scientifically sound study led the FDA to license both products, representing a milestone in EVD therapeutic field. These products can be produced relatively inexpensively and can be stockpiled. The administration of mAbs on EVD patients appears
Frontiers | Ovariectomy Impaired Hepatic Glucose and Lipid Homeostasis and Altered the Gut Microbiota in Mice With Different Diets
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Analyzing Encapsulated mRNA with LC, MS, and Calorimetry
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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, which has proven lethal in two of these subjects.
The success of the adeno-associated virus (AAV)-based product Zolgensma, approved in 2019 for the treatment of infants with spinal muscular atrophy (SMA) type 1, supported the safety and efficacy of infusing up to 1×10