RRG-003

RRG-003 is an investigational gene therapy designed to treat autosomal recessive deafness 9 (DFNB9), a form of congenital hearing loss caused by mutations in the OTOF gene. The therapy uses a dual adeno-associated virus (AAV) serotype 1 vector system to deliver a functional copy of the human otoferlin (OTOF) gene directly into the inner ear. This approach compensates for defective or missing otoferlin protein, which is essential for transmitting sound signals from the ear to the brain. Developed through collaboration between Shanghai Refreshgene Technology, Fudan University’s Eye & ENT Hospital, and Harvard Medical School, RRG-003 has shown promising results in early clinical trials—restoring hearing and speech perception in children with profound genetic deafness[1][2][3][4][5][7][8][10].

Other names

Dual recombinant adeno-associated viral vectors encoding the human otoferlin gene