other material
confidence high
sentiment positive
materiality 0.65
Graphite Bio doses first patient with nula-cel for sickle cell disease; proof-of-concept data mid-2023
LENZ Therapeutics, Inc.
- First patient dosed with nula-cel (formerly GPH101) in Phase 1/2 CEDAR trial for sickle cell disease.
- Nula-cel is a gene editing therapy designed to directly correct the SCD mutation and restore healthy hemoglobin.
- Initial proof-of-concept data from the CEDAR trial expected in mid-2023.
- FDA granted Fast Track and Orphan Drug designations for nula-cel.
- Phase 1/2 trial to enroll ~15 patients with severe SCD at multiple U.S. sites.