Clinical trial updates

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: EdiTHAL
    Code: NCT05444894
    Phase: 1/2
    Eligible patient diagnosis: TDT (adult ; ages 18 – 35)
    No. of Patients enrolled: 6 [anticipated] (Last update: 4/4/2023)
    Study Sites: Sites per country

    Anticipated completion date: : December 2025
    Scope of the Study / Aim: Safety, tolerance & efficacy of EDIT-301 infusion

  • Regulatory Information

    Regulatory Information

    Status: Not Authorised


    Additional notable points:

    • EMA: N/A
    • FDA: Rare Pediatric Disease (RPD) designation (2020); Orphan drug designation (2022)
    • MHRA: N/A

Update: 20 December 2023

The company continues to enroll and dose patients in the EdiTHAL trial for TDT.

New data

  • Data presented at the 65th ASH Annual Congress (9 – 12 December 2023) in San Diego (USA) showed that:
  • Safety:
    • Reni-cel was well-tolerated and demonstrated a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant by all patients in the RUBY and EdiTHAL trials in the 17 patients (11 in the RUBY trial / SCD ; 6 in the EdiTHAL trial / thalassaemia) that have been infused thus far.
    • After reni-cel infusion, all treated patients with >2 months follow-up demonstrated successful neutrophil engraftment within one month and platelet engraftment within 1.6 months. No serious adverse events (SAEs) related to reni-cel treatment have been reported.
  • Efficacy:
    • In the EdiTHAL trial, 5 patients with >1 month follow-up demonstrated early and robust total hemoglobin and fetal hemoglobin increases, with total hemoglobin rising above the transfusion independence threshold of 9 g/dL.



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

  • First patient dosed. The patient had successful neutrophil and platelet engraftment.
  • Data to be presented on the EDITHAL trial by year-end.

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 transfusiondependent beta thalassemia patient treated in the EdiTHAL trial.

The first treated patient (male) has had 1.5 months of follow-up. Patient 1 had successful neutrophil and platelet engraftment within 30 days of infusion. No serious adverse events occurred after EDIT-301 infusion, and no adverse events reported were related to treatment with EDIT-301.

Achieved a fetal hemoglobin fraction of 34.9% representing 4 g/dL of total hemoglobin at 1.5 months post-treatment.



Update: 31 March 2023

  • Editas Medicine remains on track to dose the first patient in the Phase 1/2 EDITHAL trial for TDT in Q1 2023.
  • The Company remains on track to present data from the EDITHAL trial by year end.


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