Rona Therapeutics Unveils Game-Changing RNAi Therapy Data for Lipoprotein(a) Reduction
In a significant scientific advancement, Rona Therapeutics showcased remarkable preclinical findings surrounding its innovative RNA interference (RNAi) therapy, named RN026. This groundbreaking therapy is aimed at attacking lipoprotein(a), a well-known independent risk factor for cardiovascular diseases. The findings were presented at the prestigious National Lipid Association (NLA) Annual Scientific Sessions held in May 2025, marking a pivotal moment in the field of cardiometabolic treatment.
Unprecedented Efficacy and Results
The preclinical studies conducted on RN026 revealed astounding results. In experiments involving transgenic mouse models, researchers observed that a single 2mg/kg subcutaneous administration of the therapy led to a 98% reduction in serum Apo(a) protein levels. This impressive effect lasted a minimum of 42 days, demonstrating the potential for sustained benefit.
Moreover, when the therapy was tested in non-human primate (NHP) studies, it achieved a staggering 99% reduction in serum lipoprotein(a) levels. Remarkably, this reduction was maintained at 95% even 98 days after the initial dose, with the serum Lp(a) levels remaining significantly lowered for up to 154 days. These results strongly suggest the possibility of an annual dosing regimen for human applications.
Precision Targeting
The design of RN026 focuses on maximizing its knock-down efficacy while minimizing off-target effects. The therapy targets multiple sites both within and outside the KIV-2 CNV region, ensuring a robust approach to inhibiting lipoprotein(a) levels effectively. This precision is critical in establishing RN026 as a safe and powerful treatment option.
Dual Advantages of the Therapy
In addition to its primary function, RN026 also demonstrated a positive secondary benefit, evidenced by a 25% reduction in LDL cholesterol levels in the NHP studies. The ongoing toxicity studies in Sprague-Dawley rats have confirmed the absence of any adverse findings, reinforcing RN026's safety profile.
Promising Horizon for Cardiovascular Therapy
As RN026 progresses in development, it stands to offer hope to the approximately 10-20% of the global population plagued by elevated serum lipoprotein(a) levels. Elevated Lp(a) is associated with several significant cardiovascular ailments, including atherosclerotic cardiovascular disease (ASCVD), aortic stenosis, and peripheral arterial disease. RN026 is positioned as a pioneering therapy that could redefine treatment paradigms in cardiovascular medicine.
About Rona Therapeutics
Rona Therapeutics is recognized globally as a leader in the development of nucleic acid-based therapies, concentrating on cardiovascular, obesity, and neurological illnesses through innovative oligonucleotide chemistry and delivery technologies. As a company dedicated to meeting the unmet needs in healthcare, Rona Therapeutics is committed to delivering first-in-class, differentiated therapies that enhance patient outcomes across various conditions.
In conclusion, the breakthrough presented by Rona Therapeutics at the NLA Scientific Sessions underscores a transformative approach to tackling lipoprotein(a) and cardiovascular health, with RN026 poised to lead the charge towards better therapeutic strategies for patients worldwide.