Editas Medicine to Present RUBY and EdiTHAL Reni-cel Data at EHA 2024

CAMBRIDGE, Mass., May 14, 2024 -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage gene editing company, has announced that two abstracts, one for an oral presentation and another for a poster presentation, have been accepted for the European Hematology Association (EHA) Hybrid Congress scheduled for June 13-16, 2024, in Madrid, Spain, and via livestream. These presentations will showcase clinical data from the RUBY and EdiTHAL trials of renizgamglogene autogedtemcel (reni-cel). 

The RUBY trial, designated Abstract #S285, will be presented orally on Saturday, June 15. This presentation will include clinical data from at least 18 patients with severe sickle cell disease, with follow-up periods ranging from 2 to 21 months. The efficacy data will cover metrics such as total hemoglobin, fetal hemoglobin, and vaso-occlusive events (VOEs). Additionally, safety data will be shared, focusing on neutrophil and platelet engraftment.

The EdiTHAL trial, designated Abstract #P1476, will be presented in a poster format on Friday, June 14. This presentation will provide clinical data from seven patients with transfusion-dependent beta thalassemia, with follow-up periods spanning 4 to 12 months. The efficacy data will outline total hemoglobin, fetal hemoglobin, and transfusion independence. Safety data will again focus on neutrophil and platelet engraftment.

Dr. Baisong Mei, Chief Medical Officer of Editas Medicine, expressed enthusiasm about the progress being made with reni-cel. "We are making significant progress with the continued development of reni-cel, and we look forward to sharing clinical data, including longer follow-up of the dosed patients from the RUBY and EdiTHAL trials next month at the European Hematology Association Congress. These data further support our belief that reni-cel has the potential to be a clinically differentiated, one-time, durable medicine that can provide life-changing clinical benefits to patients," said Dr. Mei. He also thanked the participants, their families, caregivers, clinicians, and collaborators involved in the trials.

The oral presentation, titled "Reni-cel, the first AsCas12a gene-edited cell therapy, led to hemoglobin normalization and increased fetal hemoglobin in severe sickle cell disease patients in an interim analysis of the RUBY trial," will be delivered by Dr. Rabi Hanna from the Cleveland Clinic Children’s Department of Pediatric Hematology Oncology and Blood and Marrow Transplantation. This session will take place on Saturday, June 15, 2024, from 11:30 a.m. to 12:45 p.m. CEST at Hall Velasquez, IFEMA MADRID Recinto Ferial (Fairgrounds).

The poster presentation, titled "Reni-cel, the first AsCas12a gene-edited cell therapy, shows promising preliminary results in key clinical outcomes in transfusion-dependent beta-thalassemia patients treated in the EdiTHAL trial," will be presented by Dr. Haydar Frangoul, Medical Director at Sarah Cannon Pediatric Hematology/Oncology & Cellular Therapy at TriStar Centennial. This session is scheduled for Friday, June 14, 2024, from 4:00 to 7:00 p.m. CEST at Hall 7, IFEMA MADRID Recinto Ferial (Fairgrounds).

Reni-cel is under investigation in the RUBY trial for severe sickle cell disease (NCT04853576) and the EdiTHAL trial for transfusion-dependent beta thalassemia (NCT05444894). The company plans to present further clinical updates from both trials by year-end.

Reni-cel, formerly known as EDIT-301, is an experimental gene-editing therapy targeting severe sickle cell disease and transfusion-dependent beta thalassemia. This therapy involves editing patient-derived hematopoietic stem and progenitor cells at the gamma globin gene promoters using the AsCas12a nuclease. The edited cells exhibit sustained increases in fetal hemoglobin production, potentially offering a one-time, long-lasting treatment benefit.

The RUBY trial is a single-arm, open-label, multi-center Phase 1/2 study evaluating the safety and efficacy of reni-cel in severe sickle cell disease patients. It is notable for being the first clinical trial to use AsCas12a to edit human cells successfully.

The EdiTHAL trial is similarly structured as a single-arm, open-label, multi-center Phase 1/2 study for patients with transfusion-dependent beta thalassemia, also evaluating the safety and efficacy of reni-cel.

Editas Medicine focuses on harnessing CRISPR/Cas12a and CRISPR/Cas9 to develop precision genomic medicines for serious diseases. They aim to discover, develop, and commercialize transformative treatments with long-lasting effects.

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