Drug Profile

CTX 460

Alternative Names: CTX-460

Latest Information Update: 16 Dec 2025

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At a glance

Originator CRISPR Therapeutics
Class Gene therapies
Mechanism of Action Gene expression modulators; SERPINA1 protein modulators

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity No
Available For Licensing Yes

Highest Development Phases

Preclinical Alpha 1-antitrypsin deficiency

Most Recent Events

16 Oct 2025 CTX 460 is available for licensing as of 01 Oct 2025. https://crisprtx.com/about-us/partnerships
10 Oct 2025 CRISPR Therapeutics plans to initiate clinical trial for Alpha 1-Antitrypsin Deficiency in mid-2026
10 Oct 2025 Pharmacodynamics data from a preclinical trial in Alpha-1 Antitrypsin Deficiency released by CRISPR Therapeutics

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