After raising AU$29 million (US$19.44 million) in a series A round, Rage Biotech Pty Ltd. is beginning phase I trials of its lead candidate, RB-042, an inhaled splice-switching oligonucleotide for treating chronic obstructive pulmonary disease (COPD) and other inflammatory lung diseases. Read More
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. Read More
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. Read More
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. Read More
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. Read More
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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. Read More
Tahoe Therapeutics and Alloy Therapeutics Inc. are forming a jointly seeded new company focused on developing first-in-class antibody-drug conjugates (ADCs) for patients with hard-to-treat cancers. The joint venture will advance two ADC programs directed at novel tumor targets discovered by Tahoe using its proprietary Mosaic platform and large-scale, perturbative single-cell datasets. Read More
Recursion Pharmaceuticals Inc. has prepared and tested new 1-phosphatidylinositol 3-phosphate 5-kinase (PIKfyve) inhibitors reported to be useful for the treatment of interstitial lung disease, liver, kidney and myocardial fibrosis. Read More
Work at Dark Blue Therapeutics Ltd. has led to the development of new proteolysis targeting chimera (PROTAC) compounds comprising a E3 ubiquitin ligase-binding moiety coupled to a protein ENL (MLLT1; YEATS1) and/or protein AF-9 (MLLT3; AF9)-targeting moiety through a linker. They are reported to be useful for the treatment of cancer. Read More
Bispecific T-cell engagers (TCEs) aim to combat cancer by simultaneously binding T cells and tumor cells in order to induce the first to kill the second. This approach has failed to work effectively against many solid tumors because the exogenous engager proteins do not penetrate into tumors at sufficiently high concentrations. Read More
Poly(ADP-ribose) polymerase 1 (PARP-1; ARTD1) inhibitors have been described in a Protheragen Inc. patent as potentially useful for the treatment of cancer, neurodegeneration, cardiovascular disorders, metabolic and autoimmune diseases. Read More
Eli Lilly & Co. has disclosed new glucose-dependent insulinotropic receptor (GDIR; GPR119) agonists reported to be useful for the treatment of obesity and type 2 diabetes. Read More
CD276, also known as B7-H3, is an antigen highly expressed in several cancer types, including hepatocellular carcinoma (HCC, 79%-94% expression) and closely tied to aggressiveness and poor survival, but shows very low expression in normal tissues. Increasing evidence exists indicating B7-H3 has immune inhibitory functions, thus reducing interferon levels released by T cells and suppressing cytotoxic activity of natural killer cells, thereby aiding in tumor immune evasion. Read More