Metachromatic Leukodystrophy (MLD) Archives

Takeda Drops One Hunter Syndrome Therapeutic, Puts Faith in Another

Business, Central Nervous System Diseases, Clinical Trial Endpoints, Clinical Trials, Collaborations, Hunter Syndrome, Inherited Lysosomal Storage Disorders, Metachromatic Leukodystrophy (MLD), Primary Endpoints, Quarterly results, R&D, Secondary Endpoints, Takeda

In the company’s financial results for 2021 released on May 11, Takeda announced the decision to discontinue the development of TAK-609, a therapeutic for Hunter Syndrome. Additionally, Takeda is collaborating with JCR Pharmaceuticals in another therapeutic attempt for the condition.

May 11, 2022/by BioSpace

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.

October 4, 2018/by GlobeNewswire