Editas Medicine Reports Updated Clinical Data from the RUBY Trial of Reni-cel in Patients with Severe Sickle Cell Disease at the American Society of Hematology (ASH) Annual Meeting
EDIT 12.09.2024
Drug:EDIT-301 renizgamglogene autogedtemcel (reni-cel)
Diseases:severe sickle cell disease
Date of Upcoming Event:2024-12-09
Name of Upcoming Event:American Society of Hematology (ASH) Annual Meeting
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- 01.15.2025 - 43rd Annual J.P. Morgan Healthcare Conference
- 01.13.2025 - Editas Medicine Highlights New In Vivo Preclinical Proof of Concept Data, Anticipated 2025 Key Milestones, and Three-year Strategic Priorities
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Poster presentation at ASH on
In the RUBY trial as of the data cutoff date (
Efficacy data in Patients with Severe Sickle Cell DiseasePatients were a median (range) of 9.5 (0.7–25.2) months post-reni-cel infusion, with 11 patients having >1 year follow-up. Of 28 patients, 27 were VOE-free post-reni-cel infusion. Reni-cel administration led to early, robust increases and sustained levels of total Hb and HbF. At month 6, the mean total Hb was 13.8 g/dL with a mean HbF percentage of 48.1% (n=18).
The mean percentage of F-cells increased early and was sustained at >90% from month 4 through last follow-up (n=20). MCH-F of HbF-containing red cells (F-cells) increased early, with mean value of 16.3 pg/F-cells at month 4 visit and sustained above the anti-sickling threshold of 10 pg/F-cell through last follow-up. Markers of hemolysis, including absolute reticulocyte count, indirect bilirubin, lactate dehydrogenase, and haptoglobin, improved or normalized by Month 6 and were generally maintained or improved as of last follow-up.
Sustained clinically meaningful improvements were observed in pain, physical, and social patient-reported outcome domains following treatment with reni-cel.
Safety data in Patients with Severe Sickle Cell DiseaseReni-cel was well-tolerated and demonstrated a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant by all evaluated RUBY trial patients (N=28). After reni-cel infusion, all evaluable patients (n=27) achieved successful engraftment; with median time to neutrophil engraftment of 23 days and median time to platelet engraftment of 25 days, which is important for limiting infection and bleeding risk. Two serious adverse events (SAEs) assessed by the investigators as possibly related to reni-cel treatment have been reported in the RUBY trial.
RUBY Poster Presentation Details:Title: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 TrialPresenting Author:
The poster can be accessed on the
About renizgamglogene autogedtemcel (reni-cel)Reni-cel, formerly known as EDIT-301, is an experimental gene editing medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). Reni-cel consists of patient-derived CD34+hematopoietic stem and progenitor cells edited at the gamma globin gene (HBG1andHBG2) promoters, where naturally occurring fetal hemoglobin (HbF) inducing mutations reside, by AsCas12a, a novel, proprietary, highly efficient, and specific gene editing nuclease. Red blood cells derived from reni-cel CD34+cells demonstrate a sustained increase in fetal hemoglobin production, which has the potential to provide a one-time, durable treatment benefit for people living with severe SCD and TDT.
About the RUBY TrialThe RUBY trial is a single-arm, open-label, multi-center Phase 1/2/3 study designed to assess the safety and efficacy of reni-cel in patients with severe sickle cell disease. Additional details are available onwww.clinicaltrials.gov(NCT04853576).
AboutEditas MedicineAs a leading gene editing company,
Forward-Looking StatementsThis press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘target,’’ ‘‘should,’’ ‘‘would,’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the potential of, and expectations for, the Company’s product candidates. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of clinical trials, including the RUBY trial, and clinical development of the Company’s product candidates, including reni-cel; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption “Risk Factors” included in the Company’s most recent Annual Report on Form 10-K, which is on file with the
Media and Investor Contact:Cristi Barnett (617) 401-0113cristi.barnett@editasmed.com
Source: Editas Medicine, Inc.