UX 810
Alternative Names: Microdystrophin AAV Gene therapy; UX-810Latest Information Update: 23 Jun 2022
At a glance
- Originator Solid Biosciences; Ultragenyx Pharmaceutical
- Developer Ultragenyx Pharmaceutical
- Class Gene therapies
- Mechanism of Action Dystrophin replacements; Gene transference
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- Preclinical Duchenne muscular dystrophy
Most Recent Events
- 06 Jun 2022 Preclinical trials in Duchenne muscular dystrophy in USA (Parenteral) (Ultragenyx Pharmaceutical pipeline, June 2022)