CTX 450
Alternative Names: CTX-450Latest Information Update: 23 May 2024
At a glance
- Originator CRISPR Therapeutics
- Class Gene therapies
- Mechanism of Action 5-aminolevulinate synthetase expression inhibitors
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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 Hepatic-porphyria
Most Recent Events
- 08 May 2024 Preclinical trials in Hepatic-porphyria in Switzerland (Parenteral) prior to May 2024
- 08 May 2024 Pharmacodynamics data from preclinical studies in Hepatic-porphyria were released by CRISPR Therapeutics
- 08 May 2024 CRISPR Therapeutics plans clinical trials for CTX 450 in Hepatic porphyria, in 2H of 2025