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 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 announced positive results from a two-year follow up of a stem cell gene therapy for severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID).
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.
Despite the death of one patient, bluebird bio’s Lenti-D gene therapy treatment for patients with cerebral adrenoleukodystrophy (CALD) could be a viable option for treating the potentially fatal genetic disease, researchers said.