Chengdu Chipscreen Pharmaceutical Ltd. has identified S-adenosylmethionine synthase isoform type-2 (Mat2A) inhibitors reported to be useful for the treatment of cancer.
Biohaven Therapeutics Ltd. has synthesized pyruvate kinase M2 (PKM2) activators reported to be useful for the treatment of age-related macular degeneration, amyotrophic lateral sclerosis, multiple sclerosis, retinitis pigmentosa and fibrosis.
Nikang Therapeutics Inc. has disclosed proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase-binding moiety coupled to a cyclin-dependent kinase 2 (CDK2)- and/or CDK4-targeting moiety through a linker reported to be useful for the treatment of cancer.
FGFR3 genomic alterations, including S249C as the most common, are recognized oncogenic drivers in 10%-60% of bladder cancers depending on the disease stage. Onco3r Therapeutics BV recently reported the identification of a novel series of highly potent, isoform-selective small-molecule FGFR3 inhibitors.
Triple-negative breast cancer (TNBC) is notoriously difficult to treat because it is quite aggressive and the tumors do not express the three major surface hormone receptors that can be targeted with available drugs. A potential target for treating this cancer may be glutathione peroxidase 4 (GPX4), which normally protects cells from ferroptosis, an iron-mediated form of cell death triggered by high oxidative stress.
Nectin-4 is a cell-adhesion molecule that is highly expressed in several malignancies, including bladder, colorectal, lung and breast cancers, while exhibiting minimal expression in most normal adult tissues.
Researchers from Compass Therapeutics Inc. detailed the preclinical characterization of CTX-10726, a bispecific, tetravalent antibody that simultaneously targets VEGF-A and PD-1.
A paper from Onchilles Pharma Inc. and collaborating institutions details the development of N-17350, a next-generation therapeutic elastase optimized for intratumoral delivery that targets the neutrophil elastase pathway.
Tangram Therapeutics plc has submitted a clinical trial application (CTA) to the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II trial of TGM-312 for metabolic dysfunction-associated steatohepatitis (MASH).
Glucocorticoid replacement therapy is the current standard of care for congenital adrenal hyperplasia (CAH). However, new therapeutic strategies that can better recapitulate physiological requirements and reduce morbidity and mortality among CAH patients are urgently needed. Despite the promise of gene therapy for correcting monogenic disorders, the strategies investigated to date have not yielded satisfactory results.
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