BEAM-301
Glycogen Storage Disease Type Ia (GSDIa)
PreclinicalActive
Key Facts
Indication
Glycogen Storage Disease Type Ia (GSDIa)
Phase
Preclinical
Status
Active
Company
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 Glycogen Storage Disease Type Ia (GSDIa) Drugs
| Drug | Company | Phase |
|---|---|---|
| DTX401 | Ultragenyx Pharmaceutical | Phase 3 |
| NTLA-2003 | Intellia Therapeutics | Preclinical |