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
Volunteers
Health Professionals
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).
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Principal Investigator
Sub-Investigators
- Aron Flagg, MD
- Asher Marks, MD
- Claudia Auerbach, APRN
- Farzana Pashankar, MD, MRCP, MBBS
- Juan Vasquez, MD
- Lakshmanan Krishnamurti, MD
- Mary Jane Galvin, APRN
- Mary Jane Hogan, MD, MPH, FAAP
- Niketa C Shah, MD
- Prasanna Ananth, MD, MPH
- Rozalyn Levine Rodwin, MD
- Stephanie Massaro, MD, MPH
- Stephanie Prozora, MD
- Vidya Puthenpura, MD, MHS, FAAP
- Last Updated06/13/2024
- Study HIC#2000032707