Scientists on March 31 published the first complete human genome, filling in gaps remaining after previous efforts while offering new promise in the search for clues regarding disease-causing mutations and genetic variation among the world’s 7.9 billion people.
Gene therapy development is a complex endeavor, with evolving regulations and complicated study logistics. In this article, Premier Research and Premier Consulting explore the regulatory and clinical trial landscape for gene therapy trials and offer strategies for successfully negotiating the challenges of executing these studies.
A study in Science shed new light on the global epidemic obesity by evaluating nearly 650,000 exomes from the United Kingdom, United States and Mexico and identifying those with strong implications for body mass or obesity.
Researchers with the Abramson Cancer Center of the University of Pennsylvania, led by Carl June, published results from the first U.S. Phase I trial of CRISPR-Cas9-edited T-cells in humans with advanced cancer.
The advent of increasingly more mainstream and affordable genetic testing enables powerful genetic research – but that power is directly associated with the strength and diversity of the database of genetic information available.
Alphabet’s venture capital arm GV led a $58.5 million investment to launch Verve Therapeutics, a new biotech focused on developing therapies that edit the human genome to treat heart diseases.
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.