ΔF508-CFTR is the most common CFTR mutation in cystic fibrosis (CF), which leads to destabilization of CFTR’s first nucleotide-binding domain (NBD1), contributing centrally to defective ΔF508-CFTR folding, trafficking and function.
Researchers from Arcturus Therapeutics Inc. and affiliated organizations presented preclinical data for LUNAR-CFTR, a novel mutation-agnostic, aerosolized CFTR replacement therapy for the treatment of cystic fibrosis (CF).
Rapt Therapeutics Inc. has described chemokine CCR4 receptor antagonists reported to be useful for the treatment of cancer, allergic asthma, contact dermatitis, allergic rhinitis, immunological, inflammatory, cardiovascular and metabolic disorders.
Vertex Pharmaceuticals Inc. has identified cystic fibrosis transmembrane conductance regulator (CFTR) modulators reported to be useful for the treatment of cystic fibrosis.
Work at the Agency For Science Technology & Research Bioprocessing Technology Institute has led to the identification of mitogen-activated protein kinase kinase kinase 14 (MAP3K14; NIK) inhibitors reported to be useful for the treatment of pulmonary fibrosis.
Chiesi Farmaceutici SpA has described amido cyclopropyl derivatives acting as lysophospholipid LPA1 receptor (LPAR1; EDG2) antagonists reported to be useful for the treatment of idiopathic pulmonary fibrosis (IPF).
Sarcoidosis is a multisystem disorder characterized by the formation of granulomatous inflammatory nodules mainly located in the lungs, lymphatic system, skin and eyes. In patients with pulmonary involvement, targeting IFN-γ has proven ineffective. Researchers from Baylor College of Medicine and collaborators reported on the use of nonreceptor tyrosine phosphatase Src homolog-2 domain-containing phosphatase 2 (SHP2) inhibition as a potential strategy to treat sarcoidosis-like diseases.
Japan Tobacco Inc. has divulged indazole compounds acting as hematopoietic prostaglandin D synthase (HPGDS) inhibitors reported to be useful for the treatment of chronic obstructive pulmonary disease, Duchenne muscular dystrophy, peripheral arterial disease, allergic rhinitis, sarcopenia and allergic asthma.
Transforming growth factor β1 (TGF-β1) is a key mediator of the fibrotic response, and its dysregulation drives the formation of the fibrotic niche, which impairs the capacity of tissue repair, regeneration and homeostasis.
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