Ribo expands beyond the liver with RiboPepSTAR™ – enabling targeted siRNA delivery to multiple organs
Kidney targeting
Using RiboPepSTARTM, preclinical studies demonstrate selective uptake in proximal tubular cells across models, and up to 80% target gene knockdown (KD) has been achieved, from rodents to NHPs. Physiological proof of concept was further validated in several disease models, such as type 2 diabetes model, showing robust kidney specific target-related biomarker reduction. Ribo’s first kidney-targeted drug has entered IND-enabling phase.
Cardiac targeting
A cardiac targeting conjugate resulted in sustained knockdown in heart using a mouse model. Minimal effects were observed in muscle and negligible activity in liver and kidney, confirming strong cardiac specificity.
Adipose tissue targeting
In NHPs, current delivery enabled potent and selective adipose targeting with 96% knockdown.
Together, these results underscore RiboPepSTARTM’s potential to unlock siRNA-based therapies for renal, cardiac and metabolic diseases.
In parallel, Ribo is advancing its multi target siRNA platform, enabling a single molecule to silence two or more genes simultaneously. Ribo’s dual target siRNAs achieve knockdown levels comparable to single target molecules, enabling synergistic pathway modulation and enhanced therapeutic benefit with a single therapeutic entity.
Ribo’s proprietary extra hepatic and multi targeting platforms open the door to best-in-class therapies across a broad range of disease areas - fully aligned with Ribo’s commitment to delivering next generation siRNA medicines to patients worldwide.