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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 J.P. Morgan Healthcare Conference on Tuesday, January 9 at 4:30 p.m. PST
CAMBRIDGE, Mass., Jan. 08, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (NASDAQ:EDIT), a clinical-stage genome editing company, today announced that Gilmore O'Neill, M.B., M.M.Sc., President and Chief Executive Officer, will discuss the Company's 2024 strategic priorities and anticipated milestones at the 42nd Annual J.P. Morgan Healthcare Conference on Tuesday, January 9, at 4:30 p.m. PST.
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:
"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 J.P. Morgan Healthcare Conference on Tuesday, January 9, 2024, at 4:30 p.m. PT / 7:30 p.m. ET in San Francisco, CA. A live webcast of the presentation will be available on the "Investors" section of the Editas Medicine website at www.editasmedicine.com. An archived replay will be available on the website for approximately 30 days following the presentation.
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).
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