Accelerating Splicing Modulation Therapies for Neurodegeneration with the VoyagR Platform
- Finalizing development of a lead candidate targeting HTT and PMS1 splicing with support from CIRM for IND-enabling studies in Huntington’s disease to accelerate clinical readiness and unlock therapeutic potential
- Accelerating preclinical development with refined single-molecule chemistry and new functional datasets to enhance compound validation and improve translational confidence in ReviR’s lead asset
- Expanding ReviR’s pipeline with additional splicing-modulation programs in spinal muscular atrophy and Charcot Marie-Tooth disease to broaden therapeutic reach and address unmet needs in neurodegeneration