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 & 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 – 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 – 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...
BOSTON, Mass. — Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced positive results from its once-daily vanzacaftor/tezacaftor/deutivacaftor (the “vanza triple”) program, the most comprehensive Phase 3 pivotal program ever conducted by Vertex for the treatment of cystic fibrosis (CF), a progressive, multi-organ disease caused by dysfunction of the CFTR protein. The...
BOSTON – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that in a Phase 2 proof-of-concept study, VX-864 achieved rapid, consistent and statistically significant increases in mean functional alpha-1 antitrypsin (fAAT) levels of 2.2 to 2.3 micromolar from baseline in people with alpha-1 antitrypsin deficiency (AATD) with the PiZZ genotype, across...
BOSTON – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved expanded use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis (CF) ages 6 through 11 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene...
LONDON – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced that the Australian Therapeutic Goods Administration (TGA) has approved the use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for people with cystic fibrosis (CF) ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR)...