SCD - Graphite Bio doses first patient with GPH101 for sickle cell disease
- Graphite Bio ( NASDAQ: GRPH ) is trading 2.1% higher after the company said it had finished dosing the first patient with its drug, GPH101, now called nulabeglogene autogedtemcel, in the company’s Phase 1/2 trial to treat people with sickle cell disease (SCD).
- The drug, Nula-celm is a gene editing therapy designed to directly correct the genetic mutation that causes SCD.
- "We believe nula-cel could be a definitive cure for sickle cell disease, with the potential to address all complications associated with this life-threatening disease. We look forward to reporting initial proof-of-concept data from the CEDAR trial in mid-2023," the company said.
- The trial will be conducted in about 15 patients with severe SCD. The trial is currently enrolling patients at multiple sites in the United States.
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Graphite Bio doses first patient with GPH101 for sickle cell disease