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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

To evaluate the efficacy of treatment with EDIT-301 in adult and adolescent subjects with severe sickle cell disease (SCD).

Why this Research Matters

This is a Phase 1/2 single-arm, open-label, multicenter study evaluating the safety and efficacy of a single unit dose of EDIT-301 for autologous hematopoietic stem cell transplant (HSCT) in subjects with severe SCD. Planned study subjects will be comprised of male and female adult and adolescent subjects with severe SCD, from 12 to 50 years of age, inclusive.

Who can Participate

All Ages

Patients between the ages of 12 and 50 years of age with severe Sickle Cell Disease.

Study ID

Protocol Number: 20-2626
More information available at ClinicalTrials.gov: NCT04853576

Meet the Team

Image of Principal Investigator

Christopher Mckinney, MD

Principal Investigator

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