Editas Medicine Highlights 2024 Anticipated Milestones and Strategic Priorities at the J.P. Morgan Healthcare Conference
Anticipated 2024 milestones include: present reni-cel clinical data updates mid-year and year-end, initiate the RUBY clinical trial adolescent cohort, establish in vivo preclinical proof-of-concept for an undisclosed indication, and continue to sublicense foundational IP
Strong financial position with operational runway into 2026
Company to present at the 42nd Annual
In his remarks, Dr. O’Neill will discuss several components of the Company’s strategic priorities and progress, including an unchanged focus on developing renizgamglogene autogedtemcel (reni-cel) towards biologics licensing application (BLA) and commercialization, building an in vivo pipeline, and increasing business development activities, including continuing to sublicense the Company’s foundational Cas9 and Cas12a gene editing technology.
Dr. O’Neill will also discuss the Company’s anticipated 2024 milestones:
- Continue enrollment and dosing in the RUBY and EdiTHAL clinical trials of reni-cel,
- Initiate the adolescent cohort in the RUBY trial,
- Present a substantive clinical data set of sickle cell patients with considerable clinical follow-up in the RUBY trial in mid-2024 and by year-end 2024,
- Establish in vivo preclinical proof-of-concept for an undisclosed indication, and
- Derive revenue from the Company’s foundational IP, building on the recently announced license agreements with Vertex Pharmaceuticals and
“2023 was a pivotal year for Editas as we launched and executed our focused strategy, strengthened our leadership team, and hit multiple clinical milestones to drive Editas’ transformation towards a commercial-stage company. We expect 2024 to be even more eventful as we continue to develop our potentially transformative experimental medicines,” said Dr. O’Neill.
He continued, “As a pioneer in the genome editing field, we remain focused on driving solutions for people living with serious, previously untreatable diseases by leveraging our world-class gene editing platform – in the form of developing our own clinically differentiated medicines and in the form of licenses and sublicenses to other pharmaceutical and biotechnology companies developing medicines. To be a part of this chapter of Editas’ journey is invigorating, and I look forward to what’s next.”
J.P. Morgan Healthcare Conference Webcast
Dr. O’Neill will discuss the Company’s strategic priorities and 2024 anticipated key milestones for its gene editing medicines and platform technology at the 42nd Annual
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 (HBG1 and HBG2) 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 Trial
The RUBY trial is a single-arm, open-label, multi-center Phase 1/2 study designed to assess the safety and efficacy of reni-cel in patients with severe sickle cell disease. Enrolled patients will receive a single administration of reni-cel. The RUBY trial marks the first time AsCas12a was used to successfully edit human cells in a clinical trial. Additional details are available on www.clinicaltrials.gov (NCT04853576).
About the EdiTHAL Trial
The EdiTHAL trial is a single-arm, open label, multi-center Phase 1/2 study designed to assess the safety and efficacy of reni-cel in patients with transfusion-dependent beta thalassemia. Patients will receive a single administration of reni-cel. Additional details are available on www.clinicaltrials.gov (NCT05444894).
About Editas Medicine
As a clinical-stage genome editing company,
This 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 initiation, timing, progress and results of the Company’s preclinical and clinical studies and its research and development programs, including initiating the adolescent cohort in the RUBY trial in 2024 and establishing in vivo proof of concept for an undisclosed indication in 2024, the timing for the Company’s receipt and presentation of data from its clinical trials and preclinical studies, including RUBY clinical updates in mid-2024 and by year-end 2024, the potential of, and expectations for, the Company’s product candidates, the timing or likelihood of regulatory filings and approvals, the Company’s expectations regarding commercial readiness, and the Company’s expectations regarding cash runway. 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 preclinical studies and clinical trials, including the RUBY and EdiTHAL trials, and clinical development of the Company’s product candidates, including reni-cel; availability and timing of results from preclinical studies and clinical trials; 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) 401-0113 firstname.lastname@example.org
Source: Editas Medicine, Inc.