BMPR2 mutations are the most common genetic cause of pulmonary arterial hypertension (PAH). Pulmonary artery endothelial cells (PAECs) with reduced BMPR2 expression are linked to a persistent DNA damage after reoxygenation. Forkhead box F1 (FOXF1) is a transcription factor with affinity for endothelial cells in the lung, and its reduced expression has also been associated with DNA damage in those cells and PAH.
Partners Regeneron Pharmaceuticals Inc. and Sanofi SA will file a supplemental BLA by year-end for Dupixent (dupilumab) in chronic obstructive pulmonary disease (COPD) after interim data from a second phase III trial confirmed – even bested – data from the first, demonstrating a reduction in exacerbations by 34% and improved lung function in patients with uncontrolled COPD and evidence of type 2 inflammation.
Chiesi Farmaceutici SpA has disclosed imidazole derivatives acting as TGF-β receptor type-1 (TGFBR1; ALK5; SKR4; TβR-I) inhibitors reported to be useful for the treatment of idiopathic pulmonary fibrosis (IPF).
Based on previous studies that have demonstrated the potential of growth hormone-releasing hormone receptor (GHRH-R) antagonists to modulate immune responses to bleomycin lung injury, researchers from the University of Miami and affiliated organizations aimed to evaluate the potential of the GHRH-R antagonist MIA-602 in a mouse model of rVSV-SARS-CoV-2-induced pulmonary dysfunction and heart injury.
Low expectations on Wall Street for the Pulmonary-Allergy Drugs Advisory Committee (PADAC) sit-down on Merck & Co. Inc.’s P2X3 receptor antagonist gefapixant for chronic cough (CC) translated to negative balloting by panelists. PADAC members were polled on a single question: Does the evidence demonstrate that [Merck’s] gefapixant provides a clinically meaningful benefit to adults with refractory or unexplained CC? The results came out 12 no, 1 yes.
The U.S. FDA’s Pulmonary-Allergy Drugs Advisory Committee has a lot to discuss Nov. 17, but only one voting question: Does the evidence demonstrate that Merck & Co. Inc.’s gefapixant provides a clinically meaningful benefit to adults with refractory or unexplained chronic cough?
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.
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