Dentatorubral-pallidoluysian atrophy (DRPLA) is a lethal neurodegenerative disorder caused by pathogenic expansion of CAG repeats within the atrophin-1 (ATN1) gene. As DRPLA belongs to the broader class of repeat expansion disorders (RED) that are driven by toxic gain-of-function effects, reduction or elimination of ATN1 expression is predicted to provide therapeutic benefit.
The colchicine-binding domain (CBD) of tubulin, one of the earliest identified regulatory sites, remains underexplored in oncology despite its proven success in other therapeutic areas. Drugs targeting CBD are widely used for gout, parasitic infections or actinic keratosis. However, no CBD-directed agents have reached clinical use for cancer, highlighting a significant opportunity to exploit this domain for novel anticancer strategies.
Généthon, a nonprofit laboratory created by the AFM-Téléthon, has reported its exclusive, worldwide licensing agreement with Askbio Inc., a subsidiary of Bayer AG, for the use of a patented component of AB-1009, under development by Askbio, for the treatment of Pompe disease.
Animate Biosciences Inc. has released promising preclinical results from a mouse model of pulmonary fibrosis demonstrating that two of its AI-designed therapeutic peptides significantly reduced lung fibrosis and inflammation.
Asthma is an inflammatory airway disease characterized by shortness of breath and wheezing, among others, with a varying global prevalence of 1%-18%. Both airway hyperresponsiveness and airway remodeling are two important features of the disease, where epithelial-mesenchymal transition (EMT) plays a critical role. Glucocorticoid induced 1 (GLCCI1) is a protein involved in glucocorticoid signaling that has gained interest in the field of asthma, where it has been shown to be downregulated in the lung tissues of mice with asthma and its overexpression has been found to alleviate airway remodeling.
Hainan Simcere Pharmaceutical Co. Ltd. has described N-myristoyltransferase 1 (NMT1) and NMT2 inhibitors reported to be useful for the treatment of cancer.
Guangzhou Runer Ophthalmic Biotechnology Co. Ltd. has divulged compounds acting as complement factor B (CFB) inhibitors reported to be useful for the treatment of age-related macular degeneration, diabetic retinopathy, uveitis, retinitis pigmentosa, atypical hemolytic uremic syndrome, macular edema, membranous glomerulonephritis and paroxysmal nocturnal hemoglobinuria (PNH).
Tianjin Tianyao Pharmaceuticals Co. Ltd. has identified glucocorticoid receptor (GR) agonists reported to be useful for the treatment of conjunctivitis, asthma, chronic obstructive pulmonary disease, allergic rhinitis, nasal polyps, Crohn’s disease, eczema and psoriasis.
Shenzhen Forward Pharmaceutical Co. Ltd. has synthesized quinazoline derivatives acting as GTPase KRAS G12C, G12D and G12V mutant inhibitors reported to be useful for the treatment of pancreatic cancer.
Researchers at Iama Therapeutics Srl and Italian Institute of Technology have disclosed solute carrier family 12 member 2 (SLC12A2; NKCC1) inhibitors reported to be useful for the treatment of Down syndrome.
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