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).
ASH 2020: CRISPR and Vertex’s Potential Cure for Sickle Cell Disease and More Glimmers of Hope
Acute Myeloid Leukemia (AML), American Society of Hematology (ASH) Annual Meeting, Analysts, Bristol Myers Squibb, Business, CAR-T Therapy, CD47 Inhibitors, Chronic Lymphocytic Leukemia (CLL), Clinical Studies, Clinical Trials, CRISPR, Data, Gene Therapy, Janssen, Johnson & Johnson, Large B-Cell Lymphoma, Monoclonal Antibodies, New England Journal of Medicine, R&D, Sickle Cell Disease, Therapeutics, Transfusion-Dependent Beta-Thalassemia (TDT)The American Society of Hematology (ASH) Annual Meeting & Exposition began December 5 with numerous presentations, abstracts and posters.
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.
Bluebird bio announced interim data from two different two-year clinical trials of LentiGlobin gene therapy for transfusion-dependent beta-thalassemia (TDT).
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.
Paris-based Sanofi has acquired Waltham, Mass.-based Bioverativ for about $11.6 billion.
bluebird bio announced that it expected to file three applications for regulatory approval by the end of 2019.
23andMe launches new consumer test service to check for genetic disorders
Alzheimer's, Alzheimer's Diseases, Bloom's Syndrome, Breast Cancer, Consumer Genetic Test Services, Cystic Fibrosis, DTC, FDA, Freckles, Genes, Genetic Disorders, Genetics, Hair Curliness, Health, Heart Conditions, Lactose Intolerance, Oncology, Ovarian Cancer, Sickle Cell Anemia, Tay-Sachs Disease, Tests, Thalassemia, Venture CapitalGenetics company 23andMe announced the launch of a new consumer genetic test service on Wednesday that will show whether an individual carries genes associated with 36 different disorders, such as cystic fibrosis. The launch is a major step for the company, which in 2013 was ordered by the Food and Drug Administration to stop selling […]
CAMBRIDGE, Mass. — Stocks for bluebird bio (BLUE) slid 6.5 percent Monday after the company revealed a patient who underwent an early gene study treatment for beta-thalassemia seven years ago required new treatments following a relapse. Marina Cavazzana, a professor of hematology and lead investigator for bluebird’s HGB-205 clinical study, said the patient she […]