Tides Brief | Leaderna's siRNA Drug LDR1259 Injection Receives CDE's Implied Approval for Clinical Trial in China

AboutHyperlipidemia

Hyperlipidemia is a core risk factor for the development of atherosclerotic cardiovascular disease (ASCVD). According to the latest 2026 update from the American Heart Association (AHA), cardiovascular diseases (CVD), predominantly ASCVD, remain the leading cause of death globally. Reported data indicate that in 2021, the global prevalence of CVD exceeded 600 million, with approximately 19.41 million deaths attributed to CVD, accounting for about one-third of all deaths worldwide. Statistics show that China has 478 million patients with dyslipidemia, with awareness, treatment, and control rates of only 11.7%, 10.1%, and 4.8%, respectively. In 2020, high LDL-C led to approximately 810,000 cardiovascular deaths, representing a 94% increase compared to 2010, highlighting the particularly severe burden of ASCVD. Furthermore, according to the "Chinese Guidelines for Lipid Management (2023)," the burden of ASCVD in China continues to rise, making lipid management an urgent priority.

In the management of blood lipids, the 2026 ACC/AHA Guidelines for the Management of Dyslipidemia continue to recommend low-density lipoprotein cholesterol (LDL-C) as the primary target for lipid-lowering therapy. However, growing evidence indicates that lipoprotein(a) [Lp(a)] also participates in the pathophysiological processes of atherosclerotic cardiovascular disease (ASCVD) and has become a key, independent, genetically determined risk factor. Lp(a) levels are primarily determined by genetics and are not influenced by age, sex, diet, or statin therapy. Even when patients achieve target LDL-C levels, those with elevated Lp(a) still face a persistent risk of ASCVD, constituting a significant residual cardiovascular risk.

Currently, there are no approved drugs worldwide that directly target and lower Lp(a). Therefore, developing therapeutic strategies capable of simultaneously lowering LDL-C and Lp(a) holds promise for addressing a significant clinical gap in current lipid management.

AboutLDR1259

LDR1259 Injection is a multi-target siRNA drug independently developed by Leaderna through its proprietary DIRECT technology platform. In preclinical studies, LDR1259 demonstrated the ability to simultaneously and efficiently inhibit the expression of PCSK9 and LPA mRNA, leading to sustained reductions in PCSK9 and Lp(a) protein levels, thereby continuously lowering LDL-C and Lp(a) levels in the blood. Furthermore, LDR1259 has exhibited excellent efficacy and safety in preclinical studies, with the potential for dosing every six to twelve months, offering a promising new therapeutic option for patients with hyperlipidemia!

About Leaderna

Leaderna Therapeutics Ltd. is a biotechnology company co-founded by former executives of publicly listed companies and high-level overseas talents introduced to China. Officially established in 2022, the company focuses on core technological innovations in oligonucleotides and new drug development. It has assembled a high-caliber R&D team and built efficient technology platforms for small nucleic acid modification, extrahepatic delivery, and dual-target interference. Leaderna has established a pipeline of small nucleic acid novel drugs in the field of chronic diseases, including metabolic and cardiovascular disorders, aiming to develop potential Best-in-Class or First-in-Class small nucleic acid therapeutics. Currently, the company has three small nucleic acid drug candidates in clinical development for hypertension, weight loss/metabolism, and hyperlipidemia.

Source:

1.Leaderna