SPOT mRNA03
Alternative Names: SPOT-mRNA03Latest Information Update: 30 Oct 2025
At a glance
- Originator Shanghai Siponuoyin Biotechnology
- Class Gene therapies; RNA
- Mechanism of Action Dystrophin expression modulators; RNA interference
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Orphan Drug Status
No
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Phase 0 Duchenne muscular dystrophy
Most Recent Events
- 06 Aug 2025 Phase-0 for Duchenne muscular dystrophy (In children) in China (IV) (NCT07188012)