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Search / Trial NCT06363760

A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301

Launched by EDITAS MEDICINE, INC. · Apr 9, 2024

Trial Information

Current as of July 23, 2025

Enrolling by invitation

Keywords

Sickle Cell Disease Sickle Cell Anemia Sickling Disorder Due To Hemoglobin S Beta Thalassemia Thalassemia Major Thalassemia Intermedia Hemoglobinopathies Crispr Cas12a Autologous Cd34+ Genetic Diseases, Inborn Hematologic Diseases Anemia Anemia, Hemolytic, Congenital Anemia, Hemolytic

ClinConnect Summary

This is a non-interventional, multicenter study evaluating the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion dependent b-thalassemia (TDT) who received EDIT-301 in parent studies EM-SCD-301-001 (NCT04853576) or EDIT-301-BThal-001 (NCT05444894). No investigational drug product will be administered in the LTFU study.

Gender

ALL

Eligibility criteria

  • Inclusion Criteria:
  • Participant must have received an EDIT-301 infusion as part of a clinical study.
  • Participant or legal representative/guardian (if applicable) must sign and date the informed consent form (ICF) or assent, if applicable for this long-term follow-up study.
  • Exclusion Criteria:
  • Participant is still actively enrolled in an EDIT-301 treatment study and has not yet met eligibility for long term follow-up in this study.

About Editas Medicine, Inc.

Editas Medicine, Inc. is a leading biotechnology company focused on developing transformative gene editing therapies to treat a range of serious diseases. Utilizing its proprietary CRISPR technology, Editas aims to correct genetic mutations at the source, providing innovative solutions for conditions such as inherited retinal diseases, sickle cell disease, and other genetic disorders. With a commitment to advancing the field of genomic medicine, Editas is dedicated to rigorous scientific research and clinical development, collaborating with a diverse network of partners to bring next-generation therapies from the lab to patients in need.

Locations

Minneapolis, Minnesota, United States

New York, New York, United States

Charleston, South Carolina, United States

Philadelphia, Pennsylvania, United States

Cleveland, Ohio, United States

Aurora, Colorado, United States

Saint Petersburg, Florida, United States

Ottawa, Ontario, Canada

Atlanta, Georgia, United States

Dallas, Texas, United States

Fort Worth, Texas, United States

Oakland, California, United States

Charlotte, North Carolina, United States

Columbus, Ohio, United States

New Haven, Connecticut, United States

New York, New York, United States

Nashville, Tennessee, United States

Toronto, Ontario, Canada

Patients applied

0 patients applied

Timeline

First submit

Trial launched

Trial updated

Estimated completion

Not reported