A bacteriophage is a virus that infects bacteria. They are not typically used to treat bacterial infections, but in desperate cases, they have been used to treat particularly antibiotic-resistant infections.
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).
Johnson & Johnson’s blockbuster blood cancer drug Darzalex significantly reduced the risk of disease progression or death in patients who have not been previously treated for multiple myeloma, late-stage study data showed.
Orchard Therapeutics Receives EMA PRIME Designation for OTL-300European 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.
Weeks after securing $52 million in a Series C financing, Magenta Therapeutics is eying the raising of $100 million in an initial public offering two years after it was launched by Third Rock Ventures and Atlas Venture with $48.5 million in Series A financing.
Cambridge, Massachusetts-based Magenta Therapeutics is teaming up with Ladenburg, Germany-based Heidelberg Pharma GmbH to improve curative bone marrow transplantation.
Janssen Pharmaceutical of Johnson & Johnson inked a global collaboration deal with Theravance Biopharma to develop a drug for inflammatory bowel diseases (IBD).
The U.S. FDA granted Priority Review to daratumumab as a combination treatment for patients with newly diagnosed multiple myeloma ineligible for autologous stem cell transplant.
Merck & Co. Ltd.’s drug to prevent serious infection in patients who undergo a type of stem cell transplant was approved by the U.S. FDA.
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.