An Industry-Leading Drug Discovery Platform

RNA plays a vital role in biology and represents an untapped opportunity for therapeutic intervention. At Ribometrix, we believe the time is right to unlock this potential and have built a unique drug discovery engine to deliver small-molecule therapies that prevent the production of disease-driving proteins.

The Opportunity

Protein targets have been a productive source for drug discovery and development; however, only a small fraction of disease-causing proteins have ever been successfully drugged while many are considered “undruggable” by conventional approaches. By focusing on modulating RNA biology, we circumvent these limitations and prevent the target protein from ever being produced.

Our Unique Strategy

Ribometrix has risen to the challenge of addressing “undruggable” proteins by preventing the translation of their mRNAs, leading to a reduction of the target disease-causing protein. Our platform modulates RNA biology for therapeutic effect via independent yet related strategies to develop small molecule therapeutics: direct targeting of complex RNA structures, targeting of RNA/RNA-binding protein interactions, and development of RNA degraders.

Direct RNA Targeting

Until recently, it was impossible to systematically target RNAs with small molecules due to the lack of reliable methods to consistently identify three-dimensional RNA structures or screen and optimize RNA-targeting small molecules. Ribometrix has built an industry-leading technology platform that leverages proprietary capabilities to identify and drug complex RNA structures in therapeutically compelling RNAs.

Our platform has revealed that many of these RNAs adopt unique three-dimensional structures. We validate and screen these RNA structures against diverse chemical libraries to identify druglike small molecules that bind specifically to our RNA targets. We then develop these RNA-binding small molecules into therapeutics that, through binding to mRNAs, prevent disease-causing proteins from being produced.

RNA-RBP Interactions

All aspects of RNA biology – including mRNA processing, decay, and translation – are driven by RNA-binding proteins (RBPs). With over 1,500 RBPs identified, they represent a large and diverse universe of potential drug targets. At Ribometrix, we are leveraging the potential of RBPs through individual RNA-RBP interactions and as drivers of disease-associated pathways.

Our RNA biology platform unlocks the opportunity to investigate specific RNA-RBP interactions, determining the unique sequences or structures that govern the interaction. This information, along with our drug discovery expertise, uniquely positions us to disrupt or stabilize RNA-RBP interactions with small molecules to impart specific therapeutic effects.

RNA Degraders

Our RNA degrader technology provides an opportunity to use our direct RNA targeting expertise to recruit a nuclease protein to a target mRNA. By using a “bi-functional” molecule to specifically target an RNA for degradation, we ensure that the mRNA-encoded protein is not produced. Analogous to PROTACS and protein degradation, RNA degraders represent another potent mechanism to downregulate protein expression at the mRNA level.

“Through extraordinary commitment and scientific rigor, Ribometrix is now on the verge of opening a new horizon in drug discovery — RNA modulation by small molecules.”

Mike Clayman, Chairman of the Board

Ribometrix

Ribometrix’s Capabilities

We are constantly evolving our platform by incorporating state-of-the-art capabilities

RNA Platform

Industry-leading RNA capabilities are the foundation for the discovery of small molecule modulators of RNA biology across therapeutic areas.

Experienced Team

Deep experience in RNA innovation and drug discovery, with a drive to deliver novel therapies to patients.

Partnership

Let’s Discover the Future Together

We are committed to building the industry’s leading RNA structure-based drug discovery capabilities, leveraging industry collaborations, using capital efficiently, and applying our passion for science to help people.

Partner With Us

Pipeline

Breaking Through to What’s Next

Our novel product pipeline, representing proprietary internal programs and partnered programs, carries the promise of a new paradigm of drug discovery with the goal of delivering novel therapeutics to patients in need.

Pipeline

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