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Editas Medicine, Inc. (Nasdaq: EDIT) is a clinical-stage genome editing company committed to developing transformative genomic medicines to treat serious diseases. The company leverages its proprietary CRISPR/Cas9 and CRISPR/Cas12a technology to create precise and corrective molecular modifications at the genetic level, aiming to address the root cause of various diseases. Editas Medicine has made significant strides in the development and commercialization of its genome editing platform. Currently, the company is advancing its flagship program, EDIT-301, now known as renizgamglogene autogedtemcel (reni-cel), which shows promise as a one-time, durable treatment for severe sickle cell disease (SCD) and beta thalassemia (TDT). Recent data from the RUBY and EdiTHAL trials indicate that reni-cel is well-tolerated and has demonstrated significant clinical benefits, including early correction of anemia and sustained increases in fetal hemoglobin.
Financially, Editas Medicine maintains a strong position, with cash, cash equivalents, and marketable securities amounting to $446.4 million as of September 30, 2023. The company's financial strategy is bolstered by strategic partnerships such as the recent license agreement with Vertex Pharmaceuticals and an extended collaboration with Bristol Myers Squibb. These agreements extend Editas Medicine's cash runway into 2026, supporting ongoing and future projects.
Editas Medicine is also pioneering in vivo genome editing. Recent preclinical data presented at the American Society of Gene and Cell Therapy (ASGCT) meeting showcased the company's progress in lipid nanoparticle (LNP) formulations for efficient in vivo delivery of AsCas12a mRNA, demonstrating significant scientific advancements in the field.
The company's mission is reinforced by its strong intellectual property portfolio, including exclusive licenses for CRISPR/Cas12a and CRISPR/Cas9 genome editing systems from Broad Institute and Harvard University. The company remains dedicated to translating groundbreaking genomic research into clinical applications, aiming to provide life-changing therapies for patients worldwide.
For the latest updates, including financial results, clinical trial progress, and strategic initiatives, visit the Editas Medicine website.
Editas Medicine (Nasdaq: EDIT), a clinical-stage gene editing company, has announced a conference call and webcast scheduled for August 7, 2024, at 8:00 a.m. ET. The event will cover the company's second quarter 2024 results and provide a corporate update. Interested parties can access the call via phone or webcast, with U.S. callers dialing +1-800-343-4849 and international callers using +1-203-518-9848. The webcast will be available on the company's website, and a replay will be accessible approximately one hour after the call concludes.
Editas Medicine has announced new safety and efficacy data for its gene-edited cell therapy, renizgamglogene autogedtemcel (reni-cel), from the RUBY trial, which included 18 patients with severe sickle cell disease (SCD). The data, presented at the European Hematology Association (EHA) Congress, shows that all patients treated with reni-cel experienced no vaso-occlusive events post-treatment, with follow-ups ranging up to 22.8 months.
Patients exhibited early normalization of total hemoglobin levels, averaging above 14 g/dL, and sustained improvements in fetal hemoglobin levels, exceeding 40%. The treatment was well-tolerated, with no serious adverse events reported. Editas has now dosed over 20 patients in the RUBY trial and completed adult cohort enrollment, with adolescent cohort enrollment underway.
Editas Medicine has unveiled new safety and efficacy data from the Phase 1/2 EdiTHAL trial of reni-cel in 7 patients with transfusion-dependent beta thalassemia (TDT). The gene-edited cell therapy showed promising results, with all patients maintaining hemoglobin levels above the transfusion threshold and remaining transfusion-free for 4.1 to 12.8 months post-treatment. The therapy was well-tolerated, showing a safety profile consistent with myeloablative conditioning and autologous hematopoietic stem cell transplant. These findings were presented at the European Hematology Association (EHA) Annual Congress. Reni-cel aims to be a one-time, durable treatment for TDT.
Editas Medicine (Nasdaq: EDIT) will present clinical data from two trials of renizgamglogene autogedtemcel (reni-cel) at the European Hematology Association (EHA) Hybrid Congress in Madrid, Spain, June 13-16, 2024. The RUBY trial (Phase 1/2/3) involves patients with severe sickle cell disease and will include efficacy and safety data on 18 patients with up to 21 months of follow-up. The EdiTHAL trial (Phase 1/2) targets transfusion-dependent beta thalassemia and will share data on seven patients with up to 12 months of follow-up. The presentations will include key metrics like total hemoglobin, fetal hemoglobin, and vaso-occlusive events. The company sees these updates as significant steps toward proving reni-cel as a durable, one-time treatment.
Editas Medicine showcased preclinical data at the American Society of Gene and Cell Therapy Annual Meeting, highlighting advancements in in vivo gene editing technologies. The research included the first-ever application of AsCas12a in vivo, optimized LNP delivery, and gene editing RNA guide modifications. The data supports the development of Editas Medicine's pipeline of in vivo gene editing medicines, demonstrating therapeutic promise and potential proof of concept by the end of the year.
Editas Medicine reported financial results for the first quarter of 2024, highlighting completion of adult cohort enrollment and patient dosing in the adolescent cohort for the RUBY trial for severe sickle cell disease. They are on track to present clinical data in mid-2024. The company is also enrolling patients in the EdiTHAL trial for transfusion-dependent beta thalassemia, presenting pre-clinical data at ASGCT, maintaining a strong financial position until 2026, and extending collaboration with Bristol Myers Squibb. Editas Medicine aims to establish in vivo preclinical proof-of-concept for an undisclosed indication by year-end.
Editas Medicine, Inc. (Nasdaq: EDIT) will host a conference call on May 8, 2024, to discuss first quarter 2024 results and provide a corporate update. The company will also participate in several upcoming investor events, including the Bank of America Health Care Conference and RBC Capital Markets Global Healthcare Conference.
Editas Medicine, Inc. (Nasdaq: EDIT) has extended its collaboration with Bristol Myers Squibb (NYSE: BMY) for two more years to research, develop, and commercialize alpha-beta T cell medicines for cancer and autoimmune diseases. Bristol Myers Squibb has opted into 13 programs across 11 gene targets, with potential milestone payments and royalties for Editas Medicine. The collaboration aims to create the next generation of allogeneic medicines to combat various cancers.
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