459 Studies Now Enrolling
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
Study of EDIT-301 in Patients with Severe Sickle Cell Disease (SCD)
| Sponsor: | Editas Medicine, Inc. IN004367 |
| Enrolling: | Male and Female Patients |
| IRB Number: | AAAT6997 |
| U.S. Govt. ID: | NCT04853576 |
| Contact: | Jaclyn Dosik: / jd3970@cumc.columbia.edu |
Additional Study Information:
The purpose of this study is to find out if the study medicine called EDIT-301 will help in the treatment of Severe Sickle Cell Disease (SCD) and how safe it is to be used in people. EDIT-301 (study medicine) is a new investigational therapy which uses the patient's own stem cells, modifies them by CRISPR technology, and infuses them back to the patient to treat SCD. The CRISPR technology is a method to selectively cut and modify DNA (the genetic material in all cells). This is called genome editing. Genes can be added, removed, or interrupted. In this study, the DNA of your own blood-forming cells (hematopoietic stem cells), after they are collected from you, will be modified at a special laboratory using CRISPR technology. Later, your blood-forming cells (with the changed DNA) will be given back to you so new and improved red blood cells can grow.
