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 26, 2025
Enrolling by invitation
Keywords
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.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
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
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported