Graphite Bio Shares Tank After Pausing Sickle Cell Therapy Study

  • Graphite Bio Inc GRPH voluntarily paused the Phase 1/2 CEDAR study of nulabeglogene autogedtemcel (nula-cel) for sickle cell disease (SCD) following a serious adverse event in the first patient dosed with nula-cel.
  • The company concluded that the event is likely related to the study treatment. 
  • As a result, the company will not meet its guidance for initial proof-of-concept data in mid-2023.
  • Related: BMO Capital Sees Over 400% Upside For This Small Gene-Editing Stock.
  • The decision by Graphite Bio to voluntarily pause the CEDAR study follows a serious and unexpected adverse event of prolonged low blood cell counts (pancytopenia) requiring ongoing transfusion and growth factor support in the first patient dosed with nula-cel. 
  • The patient achieved study-defined neutrophil engraftment and has shown no evidence of myelodysplasia, a rare type of blood cancer. 
  • Graphite Bio no longer expects to file an investigational new drug application for GPH102 in beta-thalassemia by mid-2024. The company is also working to identify operational efficiencies to extend its cash position to at least 2026.
  • Graphite Bio will provide a business update by the end of the first quarter of 2023.
  • Price Action: GRPH shares are down 47.70% at $1.60 during the premarket session on the last check Friday.
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