Omeros, MannKind-United Therapeutics, and Revance Therapeutics were issued Complete Response Letters from the U.S. Food and Drug Administration.
Shares of Magenta Therapeutics were down after the company announced the U.S. Food and Drug Administration placed a clinical hold on its clinical program for acute myeloid leukemia and myelodysplastic syndrome.
Bristol-Myers and Celgene's Liso-Cel Hits Mark in Multiple Cancer Studies
American Society of Hematology (ASH) Annual Meeting, B-Cell Non-Hodgkin Lymphoma, Cancer, CAR-T Therapy, Chronic Lymphocytic Leukemia (CLL), Clinical Trials, Hematopoietic Stem Cell Transplantation (HSCT), Immune Cells, R&D, Small Lymphocytic Lymphoma (SLL)Bristol-Myers Squibb announced positive results for the CAR-T therapy lisocabtagene maraleucel (liso-cel) in three studies at the American Society of Hematology Annual Meeting.
Akari Therapeutics Plc announced that the U.S. Food and Drug Administration granted Fast Track designation for nomacopan for the treatment of hematopoietic stem cell transplant associated thrombotic microangiopathy (HSCT-TMA) in pediatric patients.
Orchard Therapeutics Licenses Potentially Life-Changing MPS-I Gene Therapy Program
ADA Severe Combined Immune Deficiency (ADA-SCID), Business, Gene Therapy, Gene Transfer Technologies, Hematopoietic Stem Cell Transplantation (HSCT), Hurler syndrome (MPS I), Licensing, Neurometabolic Disorders, Stem Cell TherapyOrchard Therapeutics secured an exclusive license for an ex vivo autologous hematopoietic stem cell gene therapy program for the treatment of MPS-I developed by SR-Tiget.
Orchard Therapeutics Receives EMA PRIME Designation for OTL-300
European Medicines Agency (EMA), Gene Therapy, Hematopoietic Stem Cell Transplantation (HSCT), Metachromatic Leukodystrophy (MLD), Priority Medicines (PRIME) Designation, Rare Diseases, Transfusion-Dependent Beta-Thalassemia (TDT), Wiskott–Aldrich Syndrome (WAS), X-Linked Chronic Granulomatous Disease (X-CGD)Orchard Therapeutics announced that the European Medicines Agency granted Priority Medicines (PRIME) designation to OTL-300, an investigational autologous ex vivo lentiviral gene therapy for the treatment of transfusion-dependent beta-thalassemia.