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CTX-450

Alternative Names: CTX-450, CTX450, CTX 450
Latest Update: 2024-07-11
Latest Update Note: News Article

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

Countries in Clinic:

Active Clinical Trial Count:

Highest Development Phases

Phase 0: Porphyrias, Hepatic

Trial

Phase

Trial Status

Disease

Primary Completion Date

Probability of Success

Recent News Events

Date

Type

Title

07/11/2024

News Article

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