Akouos Inc. has received IND clearance from the FDA to initiate a first-in-human phase I/II pediatric trial of AK-OTOF, a gene therapy intended for the treatment of patients with otoferlin gene (OTOF)-mediated hearing loss.
The EMA's Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion on Sensorion SA's application seeking orphan drug designation for OTOF-GT, a gene therapy intended for the treatment of otoferlin gene-mediated hearing loss.
The therapeutic validity of RNA editing tools in vivo remains largely unknown in disease models both in terms of RNA editing efficacy and therapeutic improvement of disease-related symptoms. In recent work, scientists from the Institute of Neuroscience at the Chinese Academy of Sciences and colleagues evaluated RNA correction therapy in a mouse model that recapitulates the phenotype of human dominant-inherited deafness.
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