Benzinga | Jun 11, 2021 06:01AM EDT

Editas Medicine Highlights Presentation Of Preclinical Data Supporting Initiation Of EDIT-301 Phase 1/2 RUBY Trial For Treatment Of Sickle Cell Disease At European Hematology Association Congress

Preclinical data demonstrated robust fetal hemoglobin (HbF) induction in erythroid progeny cells with no detection of off-target editing; cells showed reduced sickling and improved rheological behavior

RUBY trial of EDIT-301 for sickle cell disease active and recruiting

CAMBRIDGE, Mass., June 11, 2021 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (NASDAQ:EDIT), a leading genome editing company, today announced preclinical data supporting the initiation of the EDIT-301 Phase 1/2 RUBY clinical trial to evaluate EDIT-301, a one-time, durable, autologous cell therapy medicine to treat sickle cell disease. EDIT-301 is the first experimental medicine generated using CRISPR/Cas12a gene editing. The Company reported the data in an oral presentation at the 26th Congress of the European Hematology Association (EHA) being held virtually.

In these preclinical studies, CD34+ cells from normal donors and sickle cell patient donors were edited at the HBG1 and HBG2 promoters with Editas-engineered highly efficient and specific AsCas12a ribonucleoprotein (RNP) at research-scale and large-scale. The data demonstrated that high levels of editing were achieved, resulting in robust fetal hemoglobin (HbF) induction in erythroid cells with no detectable off-target editing. The red blood cells derived from edited sickle cell patient CD34+ cells showed significant reduction of sickling and improved rheological behavior. In addition, data from the Company's current Good Manufacturing Practices (cGMP) clinical scale process to support EDIT-301 manufacturing demonstrated successful scale-up production, with consistent and high-level editing of the HBG1 and HBG2 promoters while maintaining high specificity and robust HbF induction. Furthermore, stable and polyclonal long-term engraftment was observed when cells manufactured from the representative scale-up process were infused into immunodeficient mice.

"High levels of editing were achieved in CD34+ cells using the highly specific, Editas-engineered Cas12a RNP, leading to potentially therapeutically relevant levels of HbF expression. These findings further support our novel approach to developing EDIT-301 as a transformative, durable medicine for the potential treatment of sickle cell disease," said Kate Zhang, Ph.D., Vice President, Biological Development, Editas Medicine. "With the current non-clinical study results and early successful tests of manufacturability, we believe our approach may yield a safer and more effective medicine, with the possibility to change the lives of people living with sickle cell disease."

The RUBY clinical trial, a Phase 1/2 trial designed to assess the safety and efficacy of EDIT-301 for the treatment of sickle cell disease, is active and recruiting. The Company remains on track to begin patient dosing in the RUBY trial by the end of 2021.