Tides Express | DNove Therapeutics, a Subsidiary of Dingxin Gene, Receives IND Approval for the World’s First Enzymatic-Process siRNA Drug DNV001 Targeting PCSK9

Apr 14, 2025 09:39 CST Updated 09:39

RRGENER

Biopharmaceutical R&D Developer

Inovatebio

Oligonucleotide Drug Developer

｜Edited by the Content Team of Zhong肽 Biochemical

April 202510DayRRGENER's Hangzhou Dinglenovo Biotechnology Co., Ltd. (hereinafter referred to as "Dinglenovo") independently developed Class I new drug DNV001 Injection has received the tacit approval for clinical trials from the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) (acceptance number: CXHL2500095). It is intended for the treatment of dyslipidemia, including adult primary hypercholesterolemia (heterozygous familial and non-familial) or mixed dyslipidemia patients characterized by elevated low-density lipoprotein cholesterol (LDL-C).

DNV001Is aGalNAc-siRNA, by knocking down in liver cellsPCSK9 mRNA, blocking from the sourcePCSK9Expression。DNV001The duration of in vivo efficacy has been extended through stability modifications. It is the first to achieve ultra-high purity in clinical batch products, with a stable and controllable process, simplifying the difficulty of subsequent process and analytical method development, optimizing the stability of scale-up production, and significantly reducing production costs. It is also a product with unit production costs low enough to meet China's medical insurance pricing under the same high-quality standards among known similar products.

Pharmaceutical and preclinical studies have shown,DNV001With excellent stability, safety, and effectiveness, it is expected to achieve the goal of long-term lipid reduction with only two injections per year in clinical practice.This product has demonstrated non-inferiority to inclisiran in both in vitro and in vivo tests (Inclisiran) The pharmacological activity can significantly reduce the levels in cellsPCSK9 mRNAAnd protein levels, dose-dependent long-term suppression in the plasma of transgenic mice and cynomolgus monkeysPCSK9Horizontal and Cynomolgus Monkey SerumLDL-CLevel. Currently, longer-term toxicology studies, combined drug toxicity studies with statins, and reproductive toxicity studies are underway.China。

AboutRRGENER

RRGENERFounded in 2020July.Possessing core delivery technologies such as AAV viral vectors and non-viral vectorsPlatform, Layout in Rare Diseases, Genetic Disorders, and Other Chronic Disease Treatment FieldsDue to the development of therapeutic drugs, committed to the development of innovative gene therapy drugs with the goal of pursuing lifelong benefits or even a cure for patients.

Source:

1.RRGENER