Research programme: RNA exon editing therapeutics - Ascidian Therapeutics/Roche
Latest Information Update: 16 May 2025
At a glance
- Originator Ascidian Therapeutics
- Class
- Mechanism of Action RNA splicing modulators
-
Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Research Neurological disorders