Dingxin Gene Reports First-in-Human Bilateral Gene Therapy for OTOF-Related Deafness in Nature Medicine

Jun 11, 2024 17:01 CST Updated 17:01

RRGENER

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June 5, 2024, ShanghaiRRGENERRRGENER, in collaboration with the Eye, Ear, Nose, and Throat Hospital of Fudan University, jointly developed the AAV1-OTOF drug, with clinical results published in the global top medical journal "Nature Medicine" (Nature Medicine) Published an article titled "Bilateral gene therapy in children with autosomal recessive deafness 9: single -arm trial results" article.

Previously, the single-ear treatment results of RRGENER's AAV1-OTOF project were published in *The Lancet* in January 2024, featured as the cover article of that issue. The current research findings pertain to the bilateral injection portion of the project.In this study, bilateral gene therapy was performed on five patients, which not only restored their hearing but also further improved their speech function and sound localization ability. This project is the world's first clinical trial of bilateral gene therapy.

In this study, none of the five patients had undergone cochlear implant surgery and were profoundly deaf. After treatment, hearing thresholds at different frequencies showed varying degrees of recovery. At 13 weeks post-treatment, the average ABR hearing recovery of all patients reached 69.4dB.

The speech recognition ability of five patients significantly improved after administration and continued to enhance over time. Additionally, the patients underwent sound source localization tests, and after administration, they were able to distinguish sounds from different directions and could listen to music and dance. In terms of safety, no dose-limiting toxicity or serious adverse events have occurred with this gene therapy so far, demonstrating favorable preliminary safety.

Zhou Rui, founder of RRGENER, stated, "We are very pleased that following the publication of the AAV1-OTOF project's research results in The Lancet earlier this year, the results of bilateral ear administration have now been published in the top medical journal, Nature Medicine. The restoration of hearing in both ears, the ability to perceive music, and identify sound sources can significantly improve patients' quality of life. We hope the AAV1-OTOF project will proceed smoothly and that more gene therapy drugs will be able to cure patients with deafness in the future."

About RRGENER

RRGENERFounded in July 2020 by a team of seasoned industry experts in the biopharmaceutical field, RRGENER focuses on the development of gene therapy products for ophthalmology, otology, metabolism, and other fields. With a goal-oriented approach, the company addresses clinical pain points to drive new drug innovation, adopting different gene delivery tools and gene therapy strategies for various application scenarios. RRGENER has successfully completed early clinical proof-of-concept for its ophthalmology and otology gene therapy pipelines, with the hope of benefiting patients sooner by providing access to effective and affordable treatments. The company is progressively building core intellectual property (IP) in both viral and non-viral delivery technologies. It has validated technical pathways and drug feasibility in relatively high-certainty R&D pipelines, solidly taking the step from 0 to 1. The next phase will revolve around the "1+X" strategy to explore new targets, new directions, and commercialization capabilities, establishing RRGENER’s competitive moat.

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