Drug Profile

Research programme: alpha-1 antitrypsin deficiency gene therapy - LENZ Therapeutics

Alternative Names: Research programme: AAT deficiency gene therapy - LENZ Therapeutics

Latest Information Update: 28 Apr 2026

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At a glance

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

28 Apr 2026 No recent reports of development identified for research development in Alpha-1-antitrypsin-deficiency in USA (Parenteral)
21 Mar 2024 LENZ Therapeutics has merged with Graphite Bio to form LENZ Therapeutics
21 Mar 2022 Early research in Alpha 1-antitrypsin deficiency in USA (Parenteral) (Graphite Bio pipeline, March 2022)

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