BEAM-302 is an investigational in vivo base-editing therapy in development for the treatment of alpha-1 antitrypsin deficiency (AATD). The candidate is administered as a single intravenous infusion of a liver-targeted lipid nanoparticle (LNP) that delivers mRNA encoding an adenine base editor together with a guide RNA designed to precisely correct the disease-causing PiZ (E342K) mutation in the SERPINA1 gene. By repairing the mutation at its native genomic locus, BEAM-302 aims to durably increase production of functional alpha-1 antitrypsin (M-AAT) while reducing circulating levels of toxic mutant Z-AAT, addressing both the lung and liver manifestations of AATD.
Disease: Alpha 1-Antitrypsin Deficiency, AATD, (NCT06389877)
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Candidate summary:
Disease/Disease category:
- Genetic metabolic diseases
Official title:
A Phase 1/2 Dose-exploration and Dose-expansion Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease and/or Liver Disease
Phases:
- Phase 1
- Phase 2
Status:
Active recruiting
Country:
- Australia
- Netherlands
- New Zealand
- United Kingdom
