Congenital hearing loss refers to hearing loss that is present before a child learns to speak. Variations in theOTOF gene, which encodes the calcium-binding protein otoferlin, are responsible for 1-8% cases of congenital, nonsyndromic hearing loss and are the leading cause of auditory neuropathy spectrum disorders. Nonsyndromic hearing loss is a partial or total loss of hearing that is not associated with other signs and symptoms. This aim of this study is to use CRISPR-Cas13 technology to perform RNA base-editing of a commonly found diseasing-causing mutation (p.Q829X) in OTOF.
Congenital hearing loss affects one in 500 newborns.
Official title:
An Open-label, Multiple-cohort, Dose-finding, Investigator-initiated Trial to Evaluate the Safety, Tolerability, and Efficacy of HG205 RNA Base-Editing Therapy in Subjects With OTOF-p.Q829X Mutation-associated Hearing Loss
The purpose of the study is to determine whether HG205 as CRISPR/Cas13 RNA base-editing therapy is safe and effective for the treatment of hearing loss caused by p.Q829X mutation in OTOF gene.
