BioSpace looks at the 10 biggest drug approvals of 2021, headlined by Biogen’s Aduhelm (aducanumab) for Alzheimer’s disease and Pfizer and BioNTech’s COVID-19 vaccine Comirnaty.
Shares of bluebird bio were down more than 24 percent after the company announced that the U.S. Food and Drug Administration placed a clinical hold on studies of the gene therapy elivaldogene autotemcel (eli-cel) for cerebral adrenoleukodystrophy (CALD).
Cambridge-based Magenta Therapeutics entered a non-exclusive research and clinical collaboration agreement with Beam Therapeutics.
Novartis AG secured Medicaid coverage for a pricey new sickle cell disease therapy in two U.S. states just weeks after winning U.S. approval, following an early campaign to convince local officials of the medicine’s value, according to a company executive and a Reuters review of public filings.
Top 10 Pipelines To Watch: 2019 Annual Report
Analysts, Ankylosing Spondylitis (AS), Annual Reports, Antiretroviral Drugs, Atopic Dermatitis (Eczema), Autoimmune Diseases, B Cells, Big Pharma, Biologics, Biopharma, Biotechnology, Blockbusters, BRCA Gene, BRCA mutation, Breakthrough Therapy Designation, Business, Cancer, CAR-T Therapy, Cells, Chemotherapy, Clinical Data, Clinical Trials, Collaborations, Crohn's Disease, Cystic Fibrosis, Drug Discovery, EU, European Medicines Agency (EMA), Fast Track Designation, FDA, FDA/Regulatory, February 2019, Future Blockbusters, Gene Editing, Generalized Myasthenia Gravis (gMG), Genomics, HIV, Human Genome, Immune Cells, Immune System, Immune-Mediated Diseases, Immunotherapy, Inflammatory Diseases, Injectables, Innovation, Integrase strand transfer inhibitors, Issue Archives, Japan, M&A, Med Ad News, Monoclonal Antibodies, Multiple myeloma, Neoantigens, Neurology, Neuromyelitis Optica Spectrum Disorder (NMOSD), New Drug Applications, Non-Nucleoside Reverse Transcriptase Inhibitor (NNRTI), Non-Small Cell Lung Cancer (NSCLC), Nonalcoholic Steatohepatitis (NASH), Nuclear Medicine, Oncology, Orphan Drug Designation, Paroxysmal Nocturnal Hemoglobinuria (PNH), Precision Medicine, Prescription Drug User Fee Act (PDUFA), Priority Medicines (PRIME) Designation, Priority Review Voucher, Product Pipelines, Proteins, Psoriatic Arthritis, R&D, Radioligands, Rare Disorders, Research, Rheumatoid Arthritis, RNA Interference (RNAi), Sickle Cell Disease, Small Molecules, Special Reports, T-Cells, Top 10 Pipelines, Top 10 Pipelines To Watch, Tumors, Ulcerative Colitis, Ultra Rare DiseasesThe return on R&D investment for leading biopharmaceutical manufacturers fell to a nine-year low while the U.S. FDA approved a record-breaking amount of novel medicines during 2018.
Cambridge, Mass.-based bluebird bio’s LentiGlobin gene therapy is increasing hemoglobin levels in anemia patients battling sickle cell disease and beta thalassemia, which reduces the need for blood transfusions in those patients.
Three Small CRISPR Biotechs That Could Cure 10,000 Diseases
Blood Diseases, Cancer, Collaborations, CRISPR/Cas9, Diseases, DNA, Eye Diseases, FDA, Gene Editing, Genetic Disorders, Genomics, Hematopoietic Stem Cells, Herpes Simplex Virus, Hunter Syndrome, Immune Cells, Inflammatory Disorders, J.P. Morgan Healthcare Conference, Liver Disease, Lung Disease, Muscle Disease, R&D, Sickle Cell Disease, Thalassemia, Transthyretin (TTR) Amyloidosis, Viral Infections, World Health OrganizationInvestor’s Business Daily looked at three small biotech companies that appear to be leading the CRISPR race.
bluebird bio announced that it expected to file three applications for regulatory approval by the end of 2019.
FDA approved Endari oral powder for patients 5 years and older with sickle cell disease to reduce severe complications associated with the blood disorder.