Radiopharm Theranostics Ltd. announced that the U.S. FDA has cleared the IND application for betabart (RV-01), its Lu177-B7H3 monoclonal antibody designed with strong affinity for the 4Ig isoform of B7-H3 that is highly expressed in tumors and not in healthy tissues.
People with the subtype of B-cell precursor acute lymphoblastic leukemia (BCP-ALL) associated with fusion of MEF2D with other genes show quite poor prognosis. Most patients fail to respond to hematopoietic stem cell transplantation or other conventional treatments. Researchers at Shanghai Jiao Tong University School of Medicine have reported a lead compound that could treat BCP-ALL.
An experimental gene therapy based on the prime editing technique could become an effective treatment for alternating hemiplegia of childhood, a severe and currently incurable rare disease. David Liu’s lab at the Broad Institute, the inventor of this gene edition methodology, together with scientists from The Jackson Laboratory, successfully reversed the effects of five mutations associated with this disorder in a mouse model.
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have discovered new heteroarylphenyl ether derivatives acting as cyclin-dependent kinase CDK8/cyclin C inhibitors. As such, they are reported to be useful for the treatment of graft-vs.-host disease, transplant rejection, multiple sclerosis, rheumatoid arthritis, atopic dermatitis, psoriasis, amyotrophic lateral sclerosis and stroke, among others.
Serine/threonine-protein kinase WNK1 inhibitors have been described in a Dania Therapeutics ApS patent as potentially useful for the treatment of cancer and hypertension.
Pelemed Co. Ltd. has prepared pyrrolotriazine derivatives acting as tyrosine-protein kinase Yes (YES1) inhibitors reported to be useful for the treatment of cancer.
Work at Libra Therapeutics Inc. has led to the identification of new mucolipin-1 (MCOLN1; TRPML1) activators reported to be useful for the treatment of metabolic diseases, infections, hematologic diseases, inflammation, cardiovascular, eye, neurological and renal disorders, among others.
The autoimmune disorder systemic lupus erythematosus (SLE) involves upregulation of IgD, and studies have shown that anti-IgD antibodies can alleviate the autoimmune disorder rheumatoid arthritis in mouse models. Building on this finding, researchers in Hefei and Changshu, China, have shown that interrupting the interaction between IgD and its receptor FcδR can reduce cytokine levels and phosphorylation of JAK2 and STAT3, suppressing the activation and proliferation of CD4+ T cells in SLE.
The sialyltransferase ST6GAL1 is upregulated in several types of cancer and its expression is particularly elevated in triple-negative breast cancer, which is extremely aggressive and associated with very poor prognosis.
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