Therapy Areas: Central Nervous System
Ribometrix Forges Collaboration with Genentech to Discover and Develop RNA-Targeted Small Molecule Therapeutics
6 January 2021 - - US-based Ribometrix, Inc has forged a strategic collaboration with US-based Genentech, a member of Switzerland's Roche Group, to discover and develop novel RNA-targeted small molecule therapeutics against several targets, the company said.

Ribometrix is focused on leveraging its RNA structural expertise and analytical capabilities to make the vast landscape of RNA, which is involved in a range of disease processes, accessible to small molecule therapeutics.

Ribometrix's platform uses multiple specialized technologies, including world-leading structural analytic capabilities, to identify 3D motifs in RNA molecules and then design small molecule candidates that bind to these motifs to produce a therapeutic effect.

Ribometrix will apply its proprietary discovery platform to identify and optimize small molecule compounds that modulate RNA function by targeting three-dimensional RNA structures.

Under the terms of the agreement, Ribometrix and Genentech will collaborate on the discovery and preclinical development of programs. Genentech will be responsible for further development and commercialization.

Genentech will pay Ribometrix a USD 25m upfront payment, and in return will receive exclusive rights to several predefined targets including an exclusive global license for the development and commercialization of molecules under this collaboration.

Ribometrix will also be eligible to receive potential milestone payments exceeding USD 1bn as well as tiered royalties on future global net sales of products that result from the collaboration.

Ribometrix's platform uses multiple specialized technologies, including world-leading structural analytic capabilities, to identify 3D motifs in RNA molecules and then design small molecule candidates that bind to these motifs to produce a therapeutic effect.

Ribometrix has shown that many RNAs implicated in disease contain structural pockets amenable to targeting with small molecules.

The potential benefits of small molecule binders of RNA over current protein and RNA-based approaches include potency, selectivity, oral bioavailability, tissue distribution and central nervous system penetration.

Ribometrix is a biotechnology company pioneering a completely new class of small molecule therapeutics that directly target and modulate the function of RNA to treat human diseases.

Ribometrix leverages its expertise in three-dimensional structural analysis of RNA to drive its discovery platform for the identification of novel small molecules that modulate the function of RNA and proteins associated with human disease.

Ribometrix completed a USD 30m series A financing in late 2018 and is headquartered in Durham, North Carolina.
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