Editas Medicine Granted FDA Regenerative Medicine Advanced Therapy Designation for Severe Sickle Cell Disease Treatment

Editas Medicine, Inc. recently announced the US FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to EDIT-301, an investigational, gene-editing medicine for the treatment of severe sickle cell disease (SCD).

“Sickle cell disease is a devastating disease that leads to anemia, pain crises, organ failure, and early death. Receiving RMAT designation for EDIT-301 for severe sickle cell disease highlights the urgent need for new treatment options for patients and supports our belief that EDIT-301 can provide life-changing clinical benefits to patients,” said Gilmore O’Neill, MB, MMSc, President and Chief Executive Officer, Editas Medicine. “I would like to thank the participants, their families, clinicians, and colleagues at collaborating institutions that contribute to the RUBY trial. We look forward to sharing further clinical updates including additional data for the trial prior to year-end.”

Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the development and review processes for promising regenerative medicine therapies. An investigational cell therapy medicine or gene editing medicine is eligible for RMAT designation if it is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the experimental medicine has the potential to address unmet medical needs for the disease or condition. Advantages of the RMAT designation include all the benefits of the fast track and breakthrough therapy designation programs, including but not limited to intensive FDA guidance on efficient and expedited drug development, possible rolling review, and priority review of the biologics license application (BLA), and FDA’s organizational commitment involving senior managers.

The FDA previously granted Orphan Drug Designation and Rare Pediatric Disease designation to EDIT-301 for the treatment of sickle cell disease and beta thalassemia.

Sickle cell disease is an inherited blood disorder caused by a mutation in the beta-globin gene that leads to polymerization of the sickle hemoglobin (HbS). In sickle cell disease, the red blood cells are misshapen in a sickle shape instead of a typical disc shape. The abnormal shape causes the red blood cells to have shortened lifespan and to block blood flow causing anemia, pain crises, organ failure, and early death. There are an estimated 100,000 people in the US currently living with sickle cell disease. Higher levels of fetal hemoglobin (HbF) inhibit HbS polymerization, thus reducing the manifestation of sickling.

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). EDIT-301 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 a highly specific and efficient proprietary engineered AsCas12a nuclease. Red blood cells derived from EDIT-301 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.

The RUBY trial is a single-arm, open-label, multi-center Phase 1/2 study designed to assess the safety and efficacy of EDIT-301 in patients with severe sickle cell disease. Enrolled patients will receive a single administration of EDIT-301. Additional details are available on www.clinicaltrials.gov (NCT#04853576).

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 more information, visit www.editasmedicine.com.