Shanghai Refreshgene Technology Co., Ltd., in collaboration with the research groups from the Eye, Ear, Nose, and Throat Hospital affiliated with Fudan University, has made significant progress in gene therapy for hearing loss. Their innovation, the RRG-OTOF Dual Vector AAV, has been granted the Rare Pediatric Disease Designation (RPDD) by the United States Food and Drug Administration (FDA). This recognition, given on November 18, 2023, acknowledges the therapeutic potential of RRG-OTOF in treating OTOF-mediated hearing loss, a challenging condition that previously had limited treatment options.
Hearing Loss Treatment

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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