Update: 30 September 2023
- Multi-year contract signed with Azzur Cleanrooms on Demand™ to support the manufacturing activities of cell medicines including EDIT-301.
Update: 30 June 2023
- Remain on track to dose 20 total SCD patients by year-end, with 19 patients enrolled to date.
- FDA has granted Orphan Drug Designation to EDIT-301 for the treatment of SCD.
Data presented at the 28th EHA Annual Congress (9 – 11 June 2023) in Frankfurt (Germany) showed positive initial safety and efficacy data from the first 4 patients with sickle cell disease (SCD) treated with EDIT-301 in the RUBY trial.
Patient 1 (male) has had 10 months of follow-up. Total hemoglobin returned to normal physiological level of 16.4g/dL at 5 months after infusion and has been maintained at this level at the 10-month follow-up. Fetal hemoglobin fraction increased from 5% at pre-infusion to 45.4% 5 months after treatment and 43.4% at the 10-month follow-up.
Patient 2 (female) has had 6 months of follow-up. Total hemoglobin reached normal physiological level of 12.7 g/dL at 5 months after infusion, and fetal hemoglobin increased from 10.8% at pre-infusion to 51.3% at the 6-month follow-up.
Patient 3 (female) and Patient 4 (male) have had 3 and 2 months of follow-up, respectively. Increases in total hemoglobin and fetal hemoglobin fractions for Patients 3 and 4 are following similar trajectories as seen in Patients 1 and 2 at the same timepoints.
All 4 patients are also free of vaso-occlusive events (VOEs) since infusion, with 2-10 months follow-up. No Serious Adverse events were reported or observed after EDIT-301 infusion.
Update: 31 March 2023
- After completing sequential dosing of the first two patients, Editas Medicine has commenced parallel patient dosing in the Phase 1/2 RUBY trial for severe SCD, and remains on track to dose 20 total SCD patients by year-end.
- The Company remains on track to present a clinical update from the RUBY trial bymid-2023.
- In December 2022, Editas Medicine announced positive safety and efficacy data from the first two patients treated in the RUBY trial, suggesting clinical proof of concept.