Rona submits IND for GalNAc-conjugated siRNA targeting obesity

Rona submits IND for GalNAc-conjugated siRNA targeting obesity

By: IPP Bureau

Last updated : September 16, 2025 4:37 pm



RN3161 is engineered to reduce fat while preserving lean mass, setting it apart from current treatment options


Rona Therapeutics, a global leader in next-generation RNAi therapeutics, announced the successful submission of its investigational therapy RN3161 to the Australian Human Research Ethics Committee (HREC).

RN3161 is a proprietary GalNAc-conjugated siRNA designed to silence the INHBE gene, representing a groundbreaking therapeutic approach for adults with overweight and obesity.

NHBE, a gene expressed in the liver, encodes Activin E, a protein that regulates fat metabolism. Research has shown that individuals with natural genetic variations in INHBE exhibit healthier fat distribution, improved cholesterol levels, and reduced risk of type 2 diabetes and cardiovascular disease—making it an attractive target for next-generation obesity therapies.

Developed through Rona’s proprietary GAIA platform, RN3161 is engineered to reduce fat while preserving lean mass, setting it apart from current treatment options. Optimized GalNAc delivery and advanced chemical modifications minimize off-target risks while enabling potent, durable activity. In preclinical studies, RN3161 achieved more than 90 percent knockdown of INHBE mRNA in non-human primates with a single low dose. Its long-lasting effect supports dosing as infrequently as once every six to twelve months, offering a major advantage for patient adherence and long-term weight management.

“Submitting RN3161 to HREC marks an important milestone in bringing forward a novel therapeutic for obesity,” said Alex M. DePaoli, MD, Chief Medical Officer of Rona Therapeutics. “This candidate has the potential to achieve weight reduction with preservation of muscle mass, while delivering meaningful metabolic benefits. Its durable activity and infrequent dosing could transform adherence and weight-loss maintenance, representing a new paradigm in obesity care.”

Stella Shi, Chief Executive Officer of Rona Therapeutics, added: “RN3161 has the potential to define a new standard of high-quality weight loss in the post–GLP-1 era. This milestone reflects our mission to reshape obesity treatment by delivering safe, effective, and durable RNAi therapies. We are proud to continue advancing our GAIA platform from bench to bedside, bringing innovative solutions to patients worldwide.”

With RN3161 as the fourth clinical candidate emerging from GAIA, Rona Therapeutics reinforces its leadership in RNAi innovation and its commitment to transforming the future of obesity and metabolic disease management.

Australian Human Research Ethics Committee Rona Therapeutics RNAi therapeutics

First Published : September 16, 2025 12:00 am