Hyloris Pharmaceuticals SA has announced the development of HY-083, a new proprietary intranasal formulation of a TRPV1 agonist to treat idiopathic rhinitis. Hyloris' treatment approach is to activate and depolarize TRPV1 receptors leading to restoration of a normal function of the nasal mucosa.
Decibel Therapeutics Inc. has received clearance from the FDA for its IND application to initiate a phase I/II trial of DB-OTO in pediatric patients, potentially in infants younger than 2 years of age.
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.
RSS
