Rona Therapeutics has filed an Investigational New Drug (IND) application with the Australian Human Research Ethics Committee for RN3161, a GalNAc-conjugated small interfering RNA (siRNA) designed to target the gene INHBE as a therapy for adults with overweight and obesity. According to the company, INHBE produces Activin E in the liver, which regulates fat metabolism, and genetic variations in INHBE are associated with more favorable fat distribution, better cholesterol levels, and lower risk of type 2 diabetes and heart disease.
The company claims that RN3161 achieved over 90% knockdown of INHBE messenger RNA in non-human primates using a single low dose. The compound is engineered with chemical modifications and optimized delivery to minimize off-target effects. Rona Therapeutics states that the long-lasting gene silencing profile of RN3161 supports dosing every six to twelve months, which could improve adherence and maintenance of weight loss.
RN3161 is the fourth clinical candidate developed through Rona’s GAIA™ platform, which focuses on RNA interference therapeutics. The company says this milestone underscores its progress in advancing therapies for obesity and other cardiometabolic diseases.
