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     193  0 Kommentare Editas Medicine Highlights 2024 Anticipated Milestones and Strategic Priorities at the J.P. Morgan Healthcare Conference - Seite 2

    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).

    About Editas Medicine
    As a clinical-stage genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas12a and CRISPR/Cas9 genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute’s Cas12a patent estate and Broad Institute and Harvard University’s Cas9 patent estates for human medicines. For the latest information and scientific presentations, please visit www.editasmedicine.com.

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    Editas Medicine Highlights 2024 Anticipated Milestones and Strategic Priorities at the J.P. Morgan Healthcare Conference - Seite 2 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 …

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