A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited CD34+ Human Hematopoietic Stem and Progenitor Cells (EDIT-301) in Subjects With Severe Sickle Cell Disease

About the study

The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult subjects with severe sickle cell disease (SCD).

Study point of contact

Editas Medicine's Clinical Trial Team


16 United States sites

3 Canada sites


18 Years - 50 Years


Phase 1/Phase 2

Study type






participation requirements

Diagnosis of severe sickle cell disease as defined by:

Documented severe SCD genotype (βS/βS, βS/β0, or βS/β+)
History of at least two severe vaso-occlusive crisis events per year requiring medical attention despite hydroxyurea or other supportive care measures in the two year-period prior to provision of informed consent

Karnofsky Performance Status ≥ 80


participation restrictions

Available 10/10 HLA-matched related donor
Prior HSCT or contraindications to autologous HSCT
Any contraindications to the use of plerixafor during the mobilization of hematopoietic stem cells (HSCs) and any contraindications to the use of busulfan and any other medicinal products required during the myeloablative conditioning, including hypersensitivity to the active substances or to any of the excipients
Unable to receive red blood cell (RBC) transfusion for any reason
Unable or unwilling to comply with standard of care changes in background medical treatment in preparation of, during, or following HSCT, including and not limited to discontinuation of hydroxyurea, voxelotor, crizanlizumab, or L-glutamine
Any history of severe cerebral vasculopathy
Inadequate end organ function
Advanced liver disease
Any prior or current malignancy or immunodeficiency disorder
Immediate family member with a known or suspected Familial Cancer Syndrome
Clinically significant and active bacterial, viral, fungal, or parasitic infection


  • Oakland, California, United States, UCSF Benioff Children's Hospital, 94609 [Recruiting]
  • Aurora, Colorado, United States, Children's Hospital Colorado, 80045 [Recruiting]
  • Atlanta, Georgia, United States, Children's Healthcare of Atlanta, 30322 [Recruiting]
  • Chicago, Illinois, United States, Ann & Robert H. Lurie Children's Hospital of Chicago, 60611 [Recruiting]
  • Jackson, Mississippi, United States, University of Mississippi Medical Center, 39216 [Recruiting]
  • New York, New York, United States, Columbia University Medical Center - Department of Pediatrics, 10032 [Recruiting]
  • New York, New York, United States, Columbia University Medical Center, 10032 [Recruiting]
  • Chapel Hill, North Carolina, United States, The University of North Carolina at Chapel Hill, 27599 [Recruiting]
  • Cleveland, Ohio, United States, University Hospitals Rainbow Babies & Children's Hospital, 44106 [Recruiting]
  • Cleveland, Ohio, United States, Cleveland Clinic, 44195 [Recruiting]
  • Columbus, Ohio, United States, Nationwide Children's Hospital, 43205 [Recruiting]
  • Philadelphia, Pennsylvania, United States, Children's Hospital of Philadelphia, 19104 [Recruiting]
  • Charleston, South Carolina, United States, Medical University of South Carolina, 29425 [Recruiting]
  • Nashville, Tennessee, United States, Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers, 37203 [Recruiting]
  • Dallas, Texas, United States, Texas Oncology - Baylor Charles A. Sammons Cancer Center, 75246 [Recruiting]
  • Fort Worth, Texas, United States, Cook Children's, 76104 [Recruiting]
  • Ottawa, Ontario, Canada, Ottawa Hospital Research Institute, K1H 8L6 [Recruiting]
  • Toronto, Ontario, Canada, Princess Margaret Cancer Centre, M5G 2M9 [Recruiting]
  • Montréal, Quebec, Canada, Centre Hospitalier Universitaire Sainte-Justine, H3T 1C5 [Recruiting]
Last updated 2023-01-10