BEAM-302
Alpha-1 Antitrypsin Deficiency (AATD)
Key Facts
About Beam Therapeutics
Beam Therapeutics is a clinical-stage biotech leader in base editing, a next-generation gene editing technology designed for precise, single-base DNA corrections without double-strand breaks. The company's mission is to deliver durable, one-time cures for serious genetic diseases, with its lead program, BEAM-101 for sickle cell disease and beta-thalassemia, now in Phase 1/2 trials. Beam's strategy leverages a fully integrated platform spanning discovery, development, and in-house GMP manufacturing to build a broad pipeline and establish dominant intellectual property in the precision genetic medicine space.
View full company profileTherapeutic Areas
Other Alpha-1 Antitrypsin Deficiency (AATD) Drugs
| Drug | Company | Phase |
|---|---|---|
| Unnamed AATD Program | AlveoGene | Pre-clinical |
| NB-101 | Nerai Biosciences | Pre-clinical |
| Undisclosed | GondolaBio | IND Enabling |
| TSRA-196 | Tessera Therapeutics | Phase 1 |
| BEAM-302 (for Beam Therapeutics) | Richmond Pharmacology | Phase 1/2 |
| KB408 | Krystal Biotech | Preclinical |
| Alpha-1 Antitrypsin (AAT) | Grifols | Marketed |
| GalNAc Program | Korro Bio | Preclinical |