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
[email protected]


4 United States sites


18 to 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


  • Aurora, Colorado, United States, Children's Hospital Colorado, 80045 [Recruiting]
  • New York, New York, United States, Columbia University Medical Center, 10032 [Recruiting]
  • Cleveland, Ohio, United States, Cleveland Clinic, 44195 [Recruiting]
  • Nashville, Tennessee, United States, Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers, 37203 [Recruiting]
Last updated 2021-10-01