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.
Vertex Pharmaceuticals Inc. has received clearance from the FDA for its IND application for VX-522, a messenger ribonucleic acid (mRNA) therapy targeted at treating the underlying cause of cystic fibrosis (CF).
