R007
Inherited mitochondrial disease (all genetically-confirmed forms)
Phase 2-readyActive
Key Facts
Indication
Inherited mitochondrial disease (all genetically-confirmed forms)
Phase
Phase 2-ready
Status
Active
Company
About Ribonova
RiboNova is a preclinical-stage biotech founded in 2018, targeting rare diseases through a novel platform focused on transfer RNA biology. The company's lead program, R007, is a Phase 2-ready small molecule for all forms of genetically-confirmed mitochondrial disease, addressing a significant unmet need in a patient population of approximately 75,000 in the U.S. Backed by venture capital and anchored at a prominent research institute, RiboNova is positioned to advance its pipeline while exploring broader applications of its tRNA-targeting platform for other diseases.
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