Research programme: citrullinemia type 1 therapeutics - iECURE
Latest Information Update: 24 Jun 2022
At a glance
- Originator University of Pennsylvania
- Developer iECURE; University of Pennsylvania
- Class Gene therapies
- Mechanism of Action Argininosuccinate synthase replacements; Gene modulators
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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
- Research Citrullinemia
Most Recent Events
- 24 May 2022 Early research in Citrullinemia in USA (Parenteral) as of May 2022 (iECURE pipeline, May 2022)