Clinical Trial Updates (SCD)

EDIT-301 (gene editing)

  • Product Information

    Product Information

    Scientific name: EDIT-301
    Brand name: Ν/Α
    RESPONSIBLE: Editas Medicine Inc.

  • Clinical Trial/Study Information

    Clinical Trial/Study Information

    Last update: 22/6/2023
    Trial Name: RUBY
    Code: NCT04853576
    Phase: 1/2
    Eligible patient diagnosis: SCD (adult; ages 18 – 50)
    No. of Patients enrolled: 40 [anticipated] (Last update: 7/6/2023)
    Study Sites: 22 Sites per country

    Anticipated completion date: August 2025
    Scope of the Study / Aim: Safety & efficacy of EDIT-301 infusion (no VOCs for 2 years post-infusion)

  • Regulatory Information

    Regulatory Information

    Status: Not Authorised


    Additional notable points:

    • EMA: N/A
    • FDA: Rare Pediatric Disease (RPD) designation (2020); Orphan Drug Designation (2023)
    • MHRA: N/A

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.

New data
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.


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