Vertex Pharmaceuticals Inc. has disclosed apolipoprotein L1 (APOL1) inhibitors reported to be useful for the treatment of focal segmental glomerulosclerosis and pancreatic cancer.
Vertex Pharmaceuticals Inc. has developed spirocyclic inhibitors of apolipoprotein L1 (APOL1) reported to be useful for the treatment of pancreatic cancer and focal segmental glomerulosclerosis.
Two years after a U.S. FDA advisory committee agreed that Celltrans Inc.’s pancreatic islet cellular therapy had a favorable risk-benefit profile, the agency has finally approved Lantidra (donislecel) for treating adults with type 1 diabetes.
Vertex Pharmaceuticals Inc. has identified 4-phenyl-2-(1H-1,2,3-triazol-4-yl)piperidin-4-ol derivatives acting as apolipoprotein L1 (APOL1) inhibitors reported to be useful for the treatment of pancreatic cancer, focal segmental glomerulosclerosis and renal disorders.
Barring truly major surprises, exagamglogene autotemcel (Exa-cel, Vertex Pharmaceuticals Inc.) is on track to become the first approved CRISPR-based gene editing therapy. It is partly in expectation of Exa-cel’s approval that the European Hematology Association (EHA) and the European Society for Bone Marrow Transplantation hosted a session on “transplantation versus gene therapy in sickle cell disease.”
Diogenx SAS raised €27.5 million (US$30.4 million) in a series A round to move a novel therapy for type 1 diabetes into clinical development. The Marseille, France-based company is building on the research of co-founder Patrick Collombat, an expert in beta-cell regeneration, who is based at the Insitute of Biology Valrose and the University Côte d’Azur, in Nice. Its lead drug candidate comprises a recombinant R-spondin protein, which acts on the Wnt/beta-catenin signal pathway to boost replication of endogenous functioning beta-cells.
Tevard Biosciences Inc. has entered into a 4-year global research collaboration with Vertex Pharmaceuticals Inc. aimed at creating new tRNA-based therapies for patients with Duchenne muscular dystrophy (DMD) caused by nonsense mutations, with options to expand into additional muscular dystrophies and a second indication.
As broader markets struggled throughout 2022, the biopharma industry’s largest and most lucrative companies ended the year in a strong position, according to BioWorld’s Biopharmaceutical Index (BBI), which showed the 22 component stocks climbed by 17.3% throughout the year. Neither the Nasdaq Biotechnology Index (NBI) nor the Dow Jones Industrial Average (DJIA) demonstrated such gains. They were down by 10.9% and 8.8%, respectively. All three indices, however, are following the same path in 2023, shooting up throughout the month of January and back down in recent weeks.
Vertex Pharmaceuticals Inc. has divulged N-(hydroxyalkyl (hetero)aryl) tetrahydrofuran carboxamides acting as sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of pain, arrhythmia, Charcot-Marie-Tooth disease, cough, urinary incontinence and multiple sclerosis.
Several recent patents from Vertex Pharmaceuticals Inc. have disclosed sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of pain, arrhythmia, Charcot-Marie-tooth disease, cough, urinary incontinence and multiple sclerosis.
