At the 2021 virtual JP Morgan Healthcare Conference, Vertex Pharmaceuticals’ chief executive officer Reshma Kewalramani said the company is looking to buy “mid- and late-stage assets.”
Skyhawk Therapeutics and Vertex Pharmaceuticals inked a strategic research collaboration and licensing deal focused on developing novel small molecules that modulate RNA splicing.
FDA Approves Trikafta, Symdeko and Kalydeco for CF Patients With Certain Rare Mutations
Approvals, Blockbusters, Children, Cystic Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), Expanded Approval, FDA, Genetic Diseases, in vitro data, New Indications, Rare Mutations, TherapeuticsThe U.S. Food and Drug Administration approved Vertex Pharmaceuticals Inc.’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko (tezacaftor/ivacaftor and ivacaftor) and Kalydeco (ivacaftor) for use in people with cystic fibrosis with certain rare mutations.
Roche acquired a portfolio of experimental cystic fibrosis therapies, including a candidate that has the potential to treat all patients with the disease.
Topline results from a 28-week Phase IIb trial show that lenabasum, a novel oral small molecule developed by Massachusetts-based Corbus Pharmaceuticals, did not reduce the rate of new pulmonary exacerbations compared with placebo in patients with cystic fibrosis (CF).
Moderna Inc. and Vertex Pharmaceuticals Incorporated announced a new strategic research collaboration and licensing agreement aimed at the discovery and development of lipid nanoparticles and mRNAs for the delivery of gene-editing therapies for the treatment of cystic fibrosis (CF).
The Social Distance Squad: Teens with Cystic Fibrosis Teach Other Teens How to Cope with COVID-19 Restrictions
Coronavirus Disease (COVID-19) Pandemic, Cystic Fibrosis, Healthcare Communications Agencies, Healthcare Communications Networks, Marketing & Advertising, Nonprofit, Outbreaks, Social Distancing, TeenagersTeens and tweens with cystic fibrosis are trying to teach other teens a thing or two about social distancing as part of The Social Distance Squad, a program developed by AREA 23, an FCB Health Network company, in partnership with five leading CF nonprofits.
Chicago-based AbbVie announced plans for a strategic collaboration to develop a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator compound licensed from the Cystic Fibrosis Foundation.
A Vertex triple-combination treatment for cystic fibrosis hit the mark in two Phase III trials, setting the stage for the company to secure approval for another treatment for the life-shortening genetic disease that affects approximately 75,000 people in North America, Europe and Australia.
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.