Advancing Rare Disease Drug Discovery

Ribozon is dedicated to  pioneering novel small molecule platforms targeting RNA repeat expansions implicated in rare neurological disorders, including Huntington's disease, spinocerebella ataxia 1, 2, 7, and Kennedy's disease (Spinal and bulbar muscular atrophy).

Cutting-Edge Assay Technologies for rna repeat rare disorders

Ribozon is committed to transforming the landscape of rare disease therapeutics through precision targeting of RNA repeat disorders. Using functional assays, our novel platform accelerates the identification of effective small molecule candidates, offering hope for patients with Huntington’s disease and other debilitating conditions.

Proprietary Novel Chemical Space

Ribozon designs novel proprietary small molecules to bind to pathogenic expanded RNA repeats selectively over healthy normal RNA repeats. Our design is based on the experiences in various drug discovery fields. We also used our own formula and equation for the efficacy estimate on our functional assay. We have discovered efficacious small molecules with proper structural scaffolds. We will continue on SAR (Structure-Activity Relationship) and expand to AI driven databases. Contact us to explore if your small molecules may be included into our platform.