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Trial intelligence / Profile preview
BEAM-302 phase 1/2 study in alpha-1 antitrypsin deficiency-associated lung and/or liver disease
NL-OMON60645Phase 2Recruiting
- Sponsor
- Beam Therapeutics
- Start
- Feb 2025
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Countries
- 1
01
Overview
Phase 2, open-label trial of BEAM-302 in alpha-1 antitrypsin deficiency (AATD) and alpha-1 antitrypsin deficiency emphysema (3 arms), sponsored by Beam Therapeutics. Primary endpoints: Rates of treatment-emergent adverse events and serious adverse events, Absolute blood levels of total AAT, Z-AAT, and M-AAT and 2 more. Started Feb 2025; status: recruiting.
02
Drugs
BEAM-302
03
Conditions
alpha-1 antitrypsin deficiency (AATD)alpha-1 antitrypsin deficiency emphysema
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