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CRISPR Therapeutics

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Lead Product(s): CTX001

Therapeutic Area: Hematology

Highest Development Status: Phase I/ Phase II Product Type: Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable May 11, 2020

Details:

CTX001 has received Orphan Drug Designation from the U.S. FDA for transfusion-dependent beta thalassemia and from the EMA for sickle cell disease and transfusion-dependent beta thalassemia .