Beam Therapeutics Announces Clearance of Investigational New Drug Application for BEAM-304 for the Treatment of Phenylketonuria (PKU) by the United States (U.S.) Food and Drug Administration
Beam Therapeutics received FDA clearance of its IND application for BEAM-304, an in vivo base editing therapy designed to treat phenylketonuria (PKU). The upcoming Phase 1/2 trial will initially evaluate safety and Phe reduction in patients with the R408W mutation, utilizing a platform-based approach to address multiple mutations within a single program.
Key figures
- Drug Name
- BEAM-304
- Phase Of Trial
- Phase 1/2
- Target phe level
- ≤ 360 µmol/L
- Patient population us
- 20,000
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