Dingxin Gene’s RRG001 intravitreal injection has successfully completed dosing of the first patient in Phase I/IIa

On December 28, 2023, Dingxin Gene’s independently developed first gene therapy product, RRG001 intravitreal injection, successfully completed dosing of the first patient in a multicenter Phase I/IIa clinical study for patients with neovascular age-related macular degeneration (nAMD). The study was carried out at the lead unit, Tianjin Medical University Eye Hospital, led by Professor Li Xiaorong. The first patient was dosed first by the team, and the subject’s postoperative ocular and systemic condition is good.

The initiation of Dingxin Gene’s first registered clinical trial marks a crucial step forward in the company’s development. After RRG001 received CDE approval for a clinical trial in November 2023, within just one and a half months, the company completed ethics review, project initiation, first-patient enrollment, and dosing—demonstrating an innovative pace. We would like to thank Professor Li’s team, research institutions, and cooperation partners including Piptech (必优特) for their hard work and strong support. The company will continue to fully advance the clinical development of this product, and looks forward to the product being launched as soon as possible to benefit patients worldwide.

About RRG001 intravitreal injection

The investigational product RRG001 intravitreal injection is an rAAV-based gene therapy independently developed by Dingxin Gene. The rAAV vector carries a gene encoding an engineered VEGF receptor Fc fusion protein. The product is delivered to the eye by subretinal injection in patients with nAMD. It transduces retinal/choroidal layer cells to achieve sustained expression of the VEGF receptor Fc fusion protein, thereby avoiding the drawbacks of traditional anti-VEGF drugs that require frequent intravitreal injections. Through a combined, innovative approach, it aims to achieve “lower dosing, single administration, and long-term benefit,” reducing safety risks while also lowering the treatment burden on patients.

Preliminary data from investigator-initiated clinical studies (IIT) have preliminarily shown that RRG001 intravitreal injection may achieve sustained clinical efficacy at a lower single-dose regimen. For the subjects enrolled in the three dose explorations conducted so far, safety and tolerability are confirmed, with no SAEs (serious adverse events).

This study is an I/IIa Phase I/IIa dose-escalation and dose-expansion clinical trial evaluating the safety and efficacy of single-dose, single-eye subretinal injection of RRG001 intravitreal injection in subjects with neovascular age-related macular degeneration (nAMD). The purpose is to assess the safety, tolerability, and preliminary efficacy of RRG001 intravitreal injection in patients with nAMD.