Researchers have ameliorated both monogenic and complex inflammation-driven diseases through transplantation of hematopoietic stem cells with an inserted IL-1 receptor antagonist (IL-1RA) gene. The team showed that in animal models the transplanted cells worked better than monoclonal antibodies to reduce symptoms in systemic autoinflammatory diseases (SAIDs), a group of childhood-onset, lifelong diseases that vary in severity depending on the underlying mutation, but can be life-threatening. Read More
Researchers from Zhejiang University and Shanghai Institute of Materia Medica of the Chinese Academy of Sciences have reported the discovery of new phosphoinositide 3-kinase α (PI3Kα) inhibitors for the treatment of cancer. Read More
Researchers from the Hospital Universitari Vall d’Hebron and colleagues have reported on the development of Hybri, a hybrid recombinant bispecific fusion protein with immunosuppressive activity that consists of the extracellular domain (ECD) of human PD-L2 fused via a linker to the ECD of human CTLA-4, further fused to the Fc domain of human IgG. Read More
Aegis Life Inc. and its parent company Entos Pharmaceuticals Inc. (Entos) have entered into a grant agreement with the Bill & Melinda Gates Foundation in the area of infectious disease. Read More
Curasight A/S has announced preclinical data demonstrating the effectiveness of Utreat (uPAR-targeted radionuclide therapy) in treating glioblastoma. Read More
Case Western Reserve University has patented conjugates consisting of a PSMA-targeting moiety covalently linked to a chelating agent comprising metals. Read More
An Agios Pharmaceuticals Inc. patent describes branched-chain-amino-acid aminotransferase, mitochondrial (BCAT2) inhibitors reported to be useful for the treatment of isovaleric acidemia, maple syrup urine disease, methylmalonic acidemia and propionic acidemia. Read More
Researchers from Structure Therapeutics Inc. recently presented details on the discovery and preclinical evaluation of two new apelin receptor (APJ, APLNR) agonists – ANPA-0073 and ANPA-0137 – being developed for the treatment of pulmonary diseases. Read More
Dtx Pharma Inc.’s Dtx-1252, an investigational small interfering RNA (siRNA) therapeutic, has been awarded U.S. orphan drug designation for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A). Read More
Research at Seoul National University has led to the identification of thiourea compounds acting as nuclear receptor RORα (RORA) agonists reported to be useful for the treatment of inflammation and metabolic diseases. Read More
Bronchiectasis is an irreversible chronic respiratory condition arising from secondary infections that presents as chronic cough with daily purulent sputum due to a permanent abnormal widening of bronchi. Read More
Alchemedicine Inc. has prepared and tested new angiotensin AT1 receptor (AGTR1) antagonists reported to be useful for the treatment of cancer, hypertension, glaucoma and renal disorders. Read More
Novartis AG has identified diaminocyclopentylpyridine derivatives acting as proprotein convertase subtilisin/kexin-type 9 (PCSK9) inhibitors and thus reported to be useful for the treatment of hypercholesterolemia, hyperlipidemia, hypertriglyceridemia, sitosterolemia, vascular inflammation, atherosclerosis, peripheral vascular and coronary heart disease, among others. Read More
Diffuse intrinsic pontine glioma (DIPG) is an almost universally fatal brain pediatric tumor and the only tumor indication where palliative radiotherapy is the current standard of care. Although chimeric antigen receptor (CAR) T-cell therapy may hold promise for treating DIPG, the elevated tumor heterogeneity and the prospect of antigen escape make the identification of additional targets crucial. Therefore, multiple targets need to be validated to facilitate a multipronged approach. Read More
Additional early-stage research and drug discovery news in brief, from: Karolinska Development, Tonix Pharmaceuticals, Transcode Therapeutics, Umecrine Cognition, Vycellix. Read More
