DNV001, a PCSK9-Targeting siRNA Therapy Requiring Only Twice-Yearly Injections, Granted IND Approval for Dyslipidemia Treatment

Apr 11, 2025 16:56 CST Updated 16:56

RRGENER

Biopharmaceutical R&D Developer

▎Reporting by the WuXi AppTec Content Team

On April 11, RRGENER announced that DNV001 Injection, a Class 1 new drug developed by its subsidiary Hangzhou Dinglexinwei Biotechnology Co., Ltd. ("Dinglexinwei"), has received clinical trial tacit approval from the Center for Drug Evaluation (CDE) of the China National Medical Products Administration.Intended for the treatment of dyslipidemia, including adult patients with primary hypercholesterolemia (heterozygous familial and non-familial) or mixed dyslipidemia characterized by elevated low-density lipoprotein cholesterol (LDL-C).。

Screenshot source:CDE Official Website

According to the public information from RRGENER, DNV001 is a GalNAc-siRNA.By knocking down PCSK9 mRNA in liver cells, the expression of PCSK9 is blocked at the source.。

Pharmaceutical and preclinical studies have shown that DNV001 has excellent stability, safety, and efficacy.Aiming to achieve the goal of long-acting lipid reduction with only two injections per year in clinical practice. This product has shown in both in vitro and in vivo tests that it can significantly reduce PCSK9 mRNA and protein levels in cells, with dose-dependent prolonged inhibition of PCSK9 levels in the plasma of transgenic mice and cynomolgus monkeys, as well as LDL-C levels in the serum of cynomolgus monkeys. Currently, researchersLonger-term toxicology studies, combined toxicology studies with statins, and reproductive toxicity studies are currently being conducted for this product.

In addition to the DNV001 injection recently approved for clinical trials,Another Gene Therapy Product by RRGENERRRG001 Intravitreal InjectionA 1/2a phase registration clinical study has been conducted for patients with neovascular age-related macular degeneration (nAMD). This isA recombinant adeno-associated virus (rAAV) gene therapy product, the rAAV vector carries the coding gene for an engineered VEGF receptor Fc fusion protein, delivered to the subretinal space of nAMD patients via subretinal injection, transducing retinal/choroidal layer cells to achieve sustained expression of the VEGF receptor Fc fusion protein.。

Moreover, the clinical research results of the gene therapy drug AAV1-OTOF, jointly developed by RRGENER and the Eye, Ear, Nose, and Throat Hospital of Fudan University, were successively published in The Lancet (The Lancet") and *Nature Medicine* ("Nature Medicine), which is intended for the treatment of autosomal recessive deafness 9 (DFNB9),It can deliver transgenes expressing functional otoferlin to the inner ear hair cells of patients, improving hearing from the root cause.。

References:

[1]China National Medical Products Administration Center for Drug Evaluation (CDE) Official Website.Retrieved Apr 11，2025, From https://www.cde.org.cn/main/xxgk/listpage/4b5255eb0a84820cef4ca3e8b6bbe20c

[2]Good News! RRGENER's Dinglexin Becomes the World’s First Enzymatic siRNA Drug Approved for Clinical Trials. RetrievedApr 11，2025, from https://mp.weixin.qq.com/s/OZrWYE9KaqqriVJ6BSiGSQ

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