Editas Medicine Touts Positive Safety, Efficacy Data From Two Sickle Cell Disease Patients

  • Editas Medicine Inc EDIT announced initial clinical data from the first two sickle cell disease (SCD) patients treated with EDIT-301 in the Phase 1/2 RUBY trial. 
  • EDIT-301 is under development for the treatment of severe sickle cell disease. The clinical data includes safety data from the first two patients and efficacy data from the first patient treated.
  • Both treated patients demonstrated successful neutrophil and platelet engraftment. 
  • Additionally, neither patient has experienced any vaso-occlusive events since treatment with EDIT-301 at five and 1.5 months follow-up, respectively.
  • Also see: Citing Small Population, Editas Medicine Pauses Work On Gene Therapy Trial For Genetic Eye Disorder.
  • At five months post-treatment, the first patient treated with EDIT-301 has a total hemoglobin of 16.4 g/dL, fetal hemoglobin (or HbF) of 45.4%, and a mean corpuscular HbF of 13.8 pg/red blood cell, exceeding the 10.0 pg/red blood cell threshold to suppress red blood cell sickling. 
  • Also, the HbF increase in the first patient was highly pan-cellular, with F cells steadily increasing to reach more than 95% of red blood cells.
  • EDIT-301 was well-tolerated. No serious adverse events occurred, and no adverse events reported were related to treatment with EDIT-301.
  • Further clinical updates are expected in mid-2023.
  • Price Action: EDIT shares are up 6% at $10.42 on the last check Tuesday.
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