Product Description
CTX450 is specifically designed to inhibit production of ALAS1 in the liver, preventing accumulation of neurotoxic aminolevulinic acid (ALA) and porphobilinogen (PBG). In preclinical studies, CTX450 showed ~70% liver editing and ~97% ALAS1 protein reduction, resulting in reduction of ALA and PBG disease biomarkers to normal levels in an AHP mouse model. (Sourced from: https://crisprtx.gcs-web.com/news-releases/news-release-details/crispr-therapeutics-highlights-asgct-oral-presentation-and)
Mechanisms of Action: Gene Therapy,ALAS1
Novel Mechanism: Yes
Modality: Gene Therapy
Route of Administration: N/A
FDA Designation: None *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: CRISPR Therapeutics
Company Location: ZUG V8 CH-6300
Company CEO: Samarth Kulkarni
Additonal Commercial Interests: None
Clinical Description
![Map of Global Clinical Trials for CTX-450](https://pryzm-maps.s3.us-west-2.amazonaws.com/666666current_maps.png)
Countries in Clinic:
Active Clinical Trial Count:
Highest Development Phases
Phase 0: Porphyrias, Hepatic
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