FDA-&-EMA-New-Drug Approvals(Mid-2020 Recap)-insert

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            Lead Product(s): CTX001

            Therapeutic Area: Hematology Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 11, 2020


            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 .