SOUTH SAN FRANCISCO, Calif. — Verge Genomics, a clinical-stage biotechnology company transforming drug discovery and development using artificial intelligence (AI) and human data, today announced the initiation of its Phase 1b proof-of-concept study of VRG50635 for the treatment of sporadic and familial forms of amyotrophic lateral sclerosis (ALS). The study...
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SAN FRANCISCO – Today, VeriSIM Life (VeriSIM) announces the launch of its pharmaceutical subsidiary, PulmoSIM Therapeutics (PulmoSIM) with the mission to address the unmet needs in the treatment of rare respiratory diseases, with an immediate emphasis on pulmonary arterial hypertension (PAH) and idiopathic pulmonary fibrosis (IPF). PulmoSIM is developing breakthrough...
BOSTON, Mass. — Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced several important advancements across its programs in immunoglobulin A Nephropathy (IgAN), APOL1-mediated kidney disease (AMKD) and autosomal dominant polycystic kidney disease (ADPKD). These updates represent significant progress toward reaching the Company’s goal of bringing forward first-in-class or best-in-class therapies that target...
BOSTON & CAMBRIDGE, Mass. – Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) and Obsidian Therapeutics announced today that the two companies have entered into a strategic research collaboration and licensing agreement focused on the discovery of novel therapies that regulate gene editing for the treatment of serious diseases. The collaboration leverages Obsidian’s cytoDRiVE®...
LONDON, UK — Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that it has reached a broad reimbursement agreement with NHS England for Vertex’s cystic fibrosis (CF) medicine ALYFTREK® (deutivacaftor/tezacaftor/vanzacaftor). This agreement comes as the National Institute for Health and Care Excellence (NICE) has issued a positive final draft recommendation for this medicine. This next-in-class triple combination...
LONDON – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted approval of the label extension for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in all patients ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance...
LONDON, UK — Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European Commission has granted approval for ALYFTREK® (deutivacaftor/tezacaftor/vanzacaftor) for the treatment of people with cystic fibrosis (CF) ages 6 years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. “Thousands of people with CF...
BOSTON, Mass. — Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced data from multiple studies demonstrating the clinical benefits of CASGEVY® (exagamglogene autotemcel) in people ages 5 years and older living with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). The results, including the first presentation of clinical data from pivotal...
LONDON – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced it has reached a new portfolio agreement with the Italian Medicines Agency, AIFA, for the reimbursement of all of Vertex’s approved medicines for the treatment of cystic fibrosis (CF), including KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor. Italian patients ages 12 years...
BOSTON – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that, in a Phase 2 proof-of-concept (POC) study in patients with APOL1-mediated focal segmental glomerulosclerosis (FSGS), VX-147 on top of standard of care achieved a statistically significant, substantial and clinically meaningful mean reduction of 47.6% in the urine protein to creatinine...