Rona Therapeutics Unveils Groundbreaking RNAi Therapy Data at NLA 2025

Rona Therapeutics Unveils Groundbreaking RNAi Therapy Data at NLA 2025



Rona Therapeutics, a prominent player in the RNA therapy landscape, shared exciting preclinical results for its novel RNA interference (RNAi) therapy, RN026, during the National Lipid Association's Annual Scientific Sessions taking place in 2025. The innovation aims to tackle Lipoprotein(a) (Lp(a)), a lipid known to elevate the risk of cardiovascular diseases, and the findings could signify a shift in treatment protocols for affected patients.

Key Findings on Efficacy and Durability



The preclinical studies demonstrated remarkable outcomes, indicating an impressive 99% reduction in Lp(a) levels in non-human primates following a single 2mg/kg subcutaneous injection of the therapy. This reduction not only showcases the therapy's potential efficacy but suggests that its effects could be sustained over an extended period, with 95% of Lp(a) reduction remaining after 98 days and a notable 80% persistence until day 154 post-administration.

In experiments involving transgenic mice, RN026 achieved a 98% decrease in serum Apo(a) protein levels, lasting at least 42 days. Such data reinforces the idea that RN026 might allow for an annual dosing regimen in humans, an appealing prospect for patients who currently manage chronic cardiovascular risks.

Precision siRNA Design



One of the cornerstones of RN026’s success lies in its sophisticated siRNA design. The therapy targets multiple regions within and around the KIV-2 CNV region, which optimizes its ability to reduce Lp(a) while minimizing off-target effects. With very low hybridization-related risks, the precision of the RNAi approach is particularly noteworthy as it bolsters the safety and effectiveness of the treatment.

Dual Benefits Observed



Interestingly, the preclinical studies also unveiled a secondary benefit of RN026: a 25% reduction in LDL-C levels in the non-human primate model. This dual action—targeting Lp(a) while simultaneously lowering LDL-C—positions RN026 as a compelling therapeutic option in the cardiovascular landscape. Importantly, repeated toxicology studies conducted on Sprague-Dawley rats reported no adverse effects, indicating that RN026 could possess a commendably safe profile for human use.

Implications for Cardiovascular Health



The potential impact of RN026 is substantial, with around 10-20% of the global population exhibiting elevated Lp(a) levels and, consequently, an increased risk of cardiovascular issues such as atherosclerotic cardiovascular disease (ASCVD) and aortic stenosis. As Rona Therapeutics progresses toward clinical trials, patients worldwide await what could be a revolutionary treatment option that brings new hope where few exist.

About Rona Therapeutics



Rona Therapeutics is renowned for its leadership in developing innovative nucleic acid-based drug platforms, focusing on treatments for cardiometabolic and neurological diseases, as well as obesity. With a commitment to creating first-in-class drugs that meet urgent therapeutic needs, Rona continues to pioneer advancements in RNA therapy, working diligently to improve outcomes for patients affected by cardiovascular diseases and beyond. The developments presented at the NLA 2025 session are just a glimpse into the transformative potential of RNAi technology in modern medicine.

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