Advancing Rare Disease Drug Discovery

Ribozon is dedicated to  pioneering novel small molecule platforms targeting RNA repeat expansions implicated in rare neurological disorders. Currently, we are focusing on CAG repeats found in Huntington's disease (HD), spinocerebella ataxia type 1, 2, 7 (SCA1, SCA2, SCA7), and Kennedy's disease (Spinal and bulbar muscular atrophy, SBMA).

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, SCA, and SBMA.

Proprietary Novel Chemical Space

Ribozon designs novel proprietary small molecules to bind to pathogenic expanded RNA repeats selectively over healthy normal RNA repeats. Our successful small molecules have met this goal by offering decent efficacy and selectivity. We are currently carrying out SAR (Structure-Activity Relationship) studies and structural optimization to find pre-clinical and clinical candidates. Contact us to explore if your small molecules may be included into our platform.