Prime Medicine Announces U.S. FDA Clearance of Investigational New Drug Application for PM647 in Alpha-1 Antitrypsin Deficiency
Prime Medicine's IND application for PM647, an in vivo Prime Editor for Alpha-1 Antitrypsin Deficiency, was cleared by the FDA, enabling first-in-human testing in the U.S. The Phase 1/2 open-label trial will start with lung-only AATD adults and expand to a liver-disease cohort after tolerability is established; roughly 100,000 Americans carry the PiZZ genotype PM647 targets.
Key figures
- Drug Name
- PM647
- Phase Of Trial
- Phase 1/2
- Us pizz patients
- 100000
- Initial data expected
- 2027
- Us europe pizz patients
- 200000
AI analysis
Red flags1 · Pro
The rest of the AI analysis, red flags and sentiment are part of Signal8 Pro.
AI-generated analysis of a public disclosure. Not investment advice; verify against the original document.