Shanghai Refreshgene Therapeutics Co., Ltd. Successfully Doses First Patient in Phase I/IIa Clinical Trial of RRG001 Intraocular Injection

On December 28, 2023, Shanghai Refreshgene Therapeutics Co., Ltd. successfully completed the first patient dosing in the Phase I/IIa multicenter clinical study of its first self-developed gene therapy product, RRG001 Intraocular Injection, for patients with neovascular age-related macular degeneration (nAMD). The first dosing was carried out at Tianjin Medical University Eye Hospital, the leading clinical site, under the leadership of its president, Professor Li Xiaorong. The subject’s postoperative ocular and systemic conditions were favorable.

The launch of Refreshgene’s first registered clinical trial marks a crucial step forward in the company’s development. After RRG001 received clinical trial approval from the CDE in November 2023, Refreshgene completed ethics approval, project initiation, first patient enrollment, and first patient dosing within just one and a half months. This demonstrates the speed of innovation. The company expressed its sincere gratitude to President Li’s team, the research institutions, Biotime, and other partners for their hard work and strong support. Moving forward, Refreshgene will continue to fully advance the clinical development of this product and looks forward to bringing it to market as soon as possible to benefit patients worldwide.

About RRG001 Intraocular Injection

RRG001 Intraocular Injection is a recombinant adeno-associated virus (rAAV) gene therapy product independently developed by Refreshgene. The rAAV vector carries a gene encoding an engineered VEGF receptor-Fc fusion protein. Delivered to the ocular fundus of nAMD patients via subretinal injection, it transduces retinal/choroidal layer cells to enable sustained expression of the VEGF receptor-Fc fusion protein. This approach avoids the drawbacks of frequent intravitreal injections required by traditional anti-VEGF therapies. Through combinational innovation, RRG001 is designed to achieve “lower dosing, single administration, and long-term benefit,” reducing safety risks while also lowering the treatment burden for patients.

Data from earlier investigator-initiated trials (IITs) have preliminarily shown that RRG001 Intraocular Injection can achieve sustained clinical efficacy at a lower single dose. Subjects in the three dose-exploration cohorts conducted to date were safe and well tolerated, with no serious adverse events (SAEs) reported.

The ongoing study is titled “A Phase I/IIa Clinical Study to Evaluate the Safety and Efficacy of Dose Escalation and Dose Expansion of a Single Unilateral Subretinal Injection of RRG001 Intraocular Injection in Subjects with Neovascular Age-Related Macular Degeneration.” The study aims to evaluate the safety, tolerability, and preliminary efficacy of RRG001 Intraocular Injection in patients with neovascular age-related macular degeneration (nAMD).