Research programme: alpha-1 antitrypsin deficiency gene therapy - LENZ Therapeutics
Alternative Names: Research programme: AAT deficiency gene therapy - LENZ TherapeuticsLatest Information Update: 26 Mar 2024
At a glance
- Originator Graphite Bio
- Developer LENZ Therapeutics
- Class Gene therapies; Stem cell therapies
- Mechanism of Action Cell replacements; Gene transference
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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
- Research Alpha 1-antitrypsin deficiency