Two independent studies applied CRISPR-based genetic editing – one to treat leukemia and the other to target myeloma – to overcome the challenges faced by CAR T cells, such as exhaustion, impaired activation and fratricide, a phenomenon in which they attack each other.
Idorsia Pharmaceuticals Ltd. has divulged macrocyclic compounds acting as cystic fibrosis transmembrane conductance regulator (CFTR) modulators reported to be useful for the treatment of cystic fibrosis.
Shenzhen Bay Laboratory has identified compounds reported to be useful for the treatment of cancer, eye, cardiovascular, metabolic, autoimmune, inflammatory, neurological and dermatological disorders.
Genescience Pharmaceuticals Co. Ltd. has synthesized sterols acting as estradiol 17-β-dehydrogenase 1 (HSD17B1; 17β-HSD1) inhibitors reported to be useful for the treatment of endometriosis.
Daewon Pharm Co. Ltd. has disclosed tricyclic derivatives acting as reversible H+/K+-ATPase inhibitors reported to be useful for the treatment of gastroesophageal reflux disease.
In cancer, Cdc37 is phosphorylated by casein kinase 2 (CK2) and then the phosphoprotein binds to various kinase ‘clients’ and to the chaperone Hsp90. Hsp90 facilitates the folding of the clients into fully active forms to drive the cell cycle. The plant-derived quinine methid triterpenoid celastrol can inhibit the interaction between Hsp90 and Cdc37 and thereby slow cancer growth by arresting the cell cycle and inducing apoptosis. However, researchers at China Pharmaceutical University China wanted to inhibit the ‘Hsp90-Cdc37-kinase’ cycle at multiple points simultaneously for greater therapeutic effect.
Checkpoint inhibitors have transformed cancer therapy by enhancing immune responses against tumors. However, their effectiveness is limited, as many patients do not respond due to the absence of a pre-existing immunity against the cancer cells. Addressing this gap requires new immunotherapies that can promote cancer-cell antigen recognition and engage multiple immune pathways to effectively reprogram the tumor microenvironment and stimulate a robust antitumor response.
Around the globe, fungal infections affect more than 1 billion people and account for several million deaths every year. They pose a particular problem in low- and middle-income countries, where antifungal drugs may be less available and, even if available, may prove ineffective because of fungal resistance. These considerations highlight the need for a next generation of antifungals.
Sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) are severe monogenic blood disorders caused by mutations in the β-globin gene (HBB), resulting in abnormal or insufficient production of adult hemoglobin (HbA). Among emerging therapeutic approaches, the reactivation of fetal hemoglobin (HbF) represents one of the most promising strategies for both conditions.
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