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Sickle Cell Disorders, Phase I-II

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

What is the purpose of this trial?

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

Contact Information

For more information about this study, including how to volunteer, contact Lakshmanan Krishnamurti

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  • Last Updated
    11/26/2024
  • Study HIC
    #2000032707