reni-cel (EDIT-301)
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Product Information
Scientific name: reni-cel
Brand name: Ν/Α
RESPONSIBLE: Editas Medicine Inc. -
Clinical Trial/Study Information
Trial Name: RUBY
Code: NCT04853576
Phase: 1/2
Eligible patient diagnosis: SCD (adult; ages 18 – 50)
No. of Patients enrolled: 45 [anticipated] (Last update: 16/4/2024)
Study Sites: 23 Sites per countryAnticipated completion date: August 2025
Scope of the Study / Aim: Safety & efficacy of reni-cel infusion (no VOCs for 2 years post-infusion) – utilizes CRISPR/Cas12a to edit the beta-globin locus and directly increase fetal hemoglobin
Update: 19 December 2024
Data presented at the 66th ASH Annual Congress (7 – 10 December 2024) in San Diego (USA) showed that:
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- 28 patients with SCD had received reni-cel by the cut-off date (29 October 2024)
- 2% had the βS/βSgenotype
- 11 patients have more than 1 year follow-up
- Post-infusion patients achieved durable normalisation of Hb (total Hb was 13.8g/dL at Month 6).
- 27/28 patients were VOC-free post-infusion
Sources: https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-reports-updated-clinical-data-ruby-trial-reni
Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Sustained Hemoglobin Normalization and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease Treated in the RUBY Trial
Update: 30 September 2024
Adolescent and adult cohort enrolment completed. Dosing continues. Additional clinical data is to be presented at ASH.
Update: 30 June 2024
Data presented at the 29th EHA Annual Congress (13 – 16 June 2024) in Madrid (Spain) showed that:
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- 18 patients with SCD had been dosed with reni-cel (cut-off date: 28 February 2024), with a mean 6.2 months follow-up (2 patients have more than 1 year follow-up).
- From Month 5, total Hb reached a mean of ≥14.4g/dL while HbF was at 48% by Month 4.
- All patients were VOC-free post-infusion until the cut-off date.
Update: 31 March 2024
- The company continues to enroll and dose adult patients and has initiated adolescent
- Announcement of data is expected in mid-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
- Regenerative Medicine Advanced Therapy (RMAT) designation (Oct 2023)
New data:
Data presented at the 65th ASH Annual Congress (9 – 12 December 2023) in San Diego (USA) showed that:
Safety:
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- 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:
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- In the RUBY trial, all treated patients are free of VOEs since reni-cel infusion. Patients with ≥5 months follow-up have maintained a normal hemoglobin level and a fetal hemoglobin level of >40%
- 10 patients with >1 month of follow-up followed a similar trajectory of total hemoglobin and fetal hemoglobin increases
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Sources: https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-granted-fda-regenerative-medicine-advanced
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.
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.
Sources: https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicineannounces-first-quarter-2023-results-and
https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicinereceives-fda-orphan-drug-designation-edit-301-0
https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicineannounces-positive-initial-edit-301-safety-and
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.