RRG-003, an adeno-associated virus (AAV) gene therapy co-developed by China’s Shanghai Dingxin Gene Technology Co., Ltd and the Eye & ENT Hospital of Fudan University, has initiated a clinical study targeting hearing loss in the country. This marks the first gene therapy trial for deafness, with the first patient expected to be enrolled next month.
Study Details
The investigator-initiated trial (ITT) is designed to assess the safety, tolerability, and preliminary efficacy of RRG-003 in treating deafness caused by mutations in the OTOF gene. The study has obtained ethical approval and aims to enroll 2-3 children. This pioneering trial underscores the potential of gene therapy in addressing genetic causes of hearing loss.
Therapy Profile
RRG-003 is being developed to significantly recover or improve hearing and speech functions in children with OTOF mutation-linked deafness. The therapy is delivered to the cochlea via local minimally invasive drug delivery, aiming to compensate for hearing defects and achieve hearing restoration. This approach represents a novel and potentially transformative treatment option for pediatric patients with genetic hearing impairments.-Fineline Info & Tech
