Thalassemia Archives

Feeling Blue: bluebird bio Cuts Staff by 30%, Prepares for FDA Decision

bluebird bio, Business, Cerebral Adrenoleukodystrophy (CALD), Cost Cutting, Cost Savings, FDA, Gene Therapy, Job Cuts, R&D, Restructuring, Thalassemia, Therapeutics, Transfusion-Dependent Beta-Thalassemia (TDT)

Following the ongoing trend in the pharmaceutical industry, bluebird bio orchestrated a restructuring that includes saving financially and cutting staff. The restructuring is designed to prolong the company’s budget as bluebird bio awaits decisions from the U.S. Food and Drug Administration (FDA).

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.