EDIT-301 is an investigational, gene editing medicine which consists of patient derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin gene promoters
Editas has received FDA’s RMAT designation for SCD therapy. (Credit: PublicDomainPictures from Pixabay)
Editas Medicine has received Regenerative Medicine Advanced Therapy (RMAT) designation from the US Food and Drug Administration (FDA) for EDIT-301, its severe sickle cell disease (SCD) therapy.
EDIT-301 is an investigational, gene editing medicine. It consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma-globin gene (HBG1 and HBG2) promoters.
The clinical-stage genome editing company is evaluating the SCD therapy in the RUBY trial.
The study is a single-arm, open-label, multi-centre Phase 1/2 study designed to assess the safety and efficacy of EDIT-301 in patients with severe SCD.
In the trial, the recruited patients will be administered with a single dose of the gene editing medicine.
Editas Medicine president and CEO Gilmore O’Neill said: “Sickle cell disease is a devastating disease that leads to anaemia, 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.
“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.”
The RMAT designation will accelerate the development and review processes of the medicine.
Previously, the FDA granted Orphan Drug Designation and Rare Paediatric Disease designation to EDIT-301 for the treatment of beta-thalassemia and sickle cell disease.
In July this year, Editas Medicine and Azzur Group announced the expansion of their multi-year contract to support the advancement of EDIT-301 through approval to commercialisation.