Further elevating its clinical promise, RRG-OTOF received the Orphan Drug Designation (ODD) from the US FDA on December 21, 2023. This distinction marks RRG-OTOF as the first Chinese independently developed in vivo gene therapy product for OTOF-mediated hearing loss to be recognized by the FDA.
PackGene Biotech, a strategic partner in this endeavor, played a crucial role by providing vital virus samples for the project. This collaboration has been instrumental in propelling the development of RRG-OTOF, contributing significantly to the advancement of gene therapies for hereditary deafness and bridging the gap from research to clinical application.
OTOF-mediated hearing loss, also known as DNFB9 congenital deafness, is an autosomal recessive disorder caused by mutations in the OTOF gene. The gene encodes otoferlin, a key protein essential for the function of cochlear hair cells in responding to sound stimuli. The dual-vector approach of RRG-OTOF, an innovative gene therapy, delivers the otoferlin gene to the cochlea’s inner hair cells. This single administration aims to restore auditory function by ensuring the long-term expression of functional otoferlin protein.
This breakthrough in gene therapy for hearing loss marks a significant step forward in treating genetic disorders and demonstrates the power of international collaboration in advancing medical science and patient care.
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