RARE Daily

FDA Grants Regenerative Medicine Advanced Therapy Designation to Editas for Sickle Cell Disease Treatment

October 17, 2023

Rare Daily Staff

The U.S. Food and Drug Administration granted Regenerative Medicine Advanced Therapy designation to Editas Medicine for EDIT-301, an investigational gene editing medicine, for the treatment of severe sickle cell disease.

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. 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 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.

Editas is studying EDIT0301 in the RUBY trial, a single-arm, open-label, multi-center phase 1/2 study designed to assess its safety and efficacy in patients with severe sickle cell disease. Enrolled patients will receive a single administration of EDIT-301.

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 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,” Gilmore O’Neill, president and CEO, Editas Medicine.

Photo: Gilmore O’Neill, president and CEO, Editas Medicine

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