TMS Icon
Clinical trial updates

reni-cel (EDIT-301)

Less than a minute
  • Product Information

    Product Information

    Scientific name: reni-cel
    Brand name: Ν/Α
    RESPONSIBLE: Editas Medicine Inc

  • Clinical Trial/Study Information

    Clinical Trial/Study Information

    Trial Name: EdiTHAL
    Code: NCT05444894
    Phase: 1/2
    Eligible patient diagnosis: TDT (adult; ages 18 – 35)
    No. of Patients enrolled: 9 [anticipated] (Last update: 16/4/2024)
    Study Sites: 8 Sites per country

    Anticipated completion date: : December 2025
    Scope of the Study / Aim: Safety, tolerance & efficacy of reni-cel infusion – utilizes CRISPR/Cas12a to edit the beta-globin locus and directly increase fetal hemoglobin

  • 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: 19 December 2024

Clinical data to be announced by year-end.

Sources: https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-announces-third-quarter-2024-results-and
https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-announces-progress-towards-2024-goals-including

 

Update: 30 September 2024

Adult cohort enrolment completed. Dosing continues. Additional clinical data is to be presented at ASH.

Source: https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-announces-second-quarter-2024-results-and

 

Update: 30 June 2024

Data presented at the 29th EHA Annual Congress (13 – 16 June 2024) in Madrid (Spain) showed that:

    • 7 patients with TDT had been dosed with reni-cel (cut-off date: 9 February 2024), with a mean 5.8 months follow-up.
    • By Month 6, total Hb reached a mean of 12.8g/dL while HbF increased early and reached a mean 8.9g/dL by Month 3.
    • All patients maintained transfusion independence for up to 9.9 months.

 

 

 

 

 

Sources: Reni-Cel, the First Ascas12a Gene-Edited Cell Therapy, Shows Promising Preliminary Results in Key Clinical Outcomes Intransfusion-Dependent Beta-Thalassemia Patients Treated in the EdiThal Trial

 

Update: 31 March 2024

  • The Company remains on-track to present additional clinical data from the EdiTHAL trial in mid-2024 and further data by year-end 2024.

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Sources: https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-announces-fourth-quarter-and-full-year-2023-0
April 2024 Corporate Presentation

 

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.

Sources: https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-announces-third-quarter-2023-results-and
https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-present-clinical-data-ruby-and-edithal-trials
https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-announces-new-edit-301-safety-and-efficacy-data

 

Update: 30 September 2023

  • Multi-year contract signed with Azzur Cleanrooms on Demand™ to support the manufacturing activities of cell medicines including EDIT-301.

Source: https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-and-azzur-group-expand-partnership-accelerate    

 

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.

Sources: https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicineannounces-first-quarter-2023-results-and
https://www.editasmedicine.com/wp-content/uploads/2023/06/EDIT-301_12-JUN2023_Webinar-FINAL.pdf
https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicineannounces-positive-initial-edit-301-safety-and

 

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.

Source: https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicineannounces-fourth-quarter-and-full-year-2022 

Less than a minute
Back to top button