The mRNA technology used in vaccines against viral infections could also be developed for cancer therapies. A group of scientists has designed a circular RNA (circRNA) encapsulated in lipid nanoparticles (LNPs) that acts in the mitochondria of tumor cells through the protein gasdermin-D (GSDMD) and reduces adenocarcinoma. The work was published on Oct. 16, 2023, in Nature Cancer. Read More
African Americans are 10 times more likely to develop chronic kidney disease (CKD) than Americans of European descent in part due to inheritance of one of two high-risk (HR) APOL1 variants (G1/G2, not G0) that are only present in people of West African or Caribbean ancestry. By contrast, these two APOL1 HR variants confer a beneficial resistance to otherwise lethal trypanosomiasis, which is caused by a pathogen endemic to West Africa, but still this ultimately results in a 4-fold increased risk for end-stage kidney disease (ESKD). Read More
Silo Pharma Inc. has announced promising data from a preclinical study investigating the binding affinity and optimization of SPU-21 liposomal joint homing peptide in human synovial tissue surrounding joints and tendons. Read More
Vividion Therapeutics Inc. has disclosed VVD-065, a novel, first-in-class, allosteric molecular glue of the KEAP1-CUL3 E3-ligase complex, for the potential treatment of NRF2-activated cancers. NRF2 (nuclear factor erythroid 2-related factor 2) is a significant mediator of antioxidant response. Read More
Structure-guided lead optimization at Merck & Co. Inc. has led to the discovery of MK-1084 as a highly potent and selective KRAS G12C-GDP inhibitor. As reported at the recent AACR-NCI-EORTC conference, the candidate demonstrated an excellent off-target and ion channel profile, with low potential for bile salt export pump (BSEP) inhibition. Read More
Caribou Biosciences Inc. has received FDA clearance of its IND application for CB-012, an allogeneic anti-C-type lectin-like molecule-1 (anti-CLL-1) chimeric antigen receptor (CAR) T-cell therapy. CLL-1 is highly expressed on acute myeloid leukemia (AML) cells and leukemic stem cells, but it is not expressed on hematopoietic stem cells. Read More
The University of Minnesota has disclosed monocarboxylate transporter (MCT1) inhibitors reported to be useful for the treatment of cancer, graft-vs.-host disease, nonalcoholic steatohepatitis, obesity and diabetes. Read More
Generation Bio Co. has announced a breakthrough in its nonviral genetic medicine platform with the development of a proprietary, novel DNA called immune-quiet DNA (iqDNA).
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Suzhou Zelgen Biosciences Co. Ltd. has synthesized GTPase KRAS (G12D mutant) inhibitors reported to be useful for the treatment of pancreatic cancer. Read More
Alexion Pharmaceuticals Inc. has identified complement C1s subcomponent inhibitors reported to be useful for the treatment of immune thrombocytopenia purpura, lupus nephritis, amyotrophic lateral sclerosis, Guillain Barré syndrome, Huntington’s disease, rheumatoid arthritis, traumatic brain injury, and autoimmune hemolytic anemia, among others. Read More
Research at Aurigene Oncology Ltd. has led to the discovery of a mucosa-associated lymphoid tissue lymphoma translocation protein 1 (MALT1) inhibitor AU-MALT1-01 for the potential treatment of B-cell lymphomas including diffuse large B-cell lymphoma (DLBCL). Read More
Ventus Therapeutics US Inc. has divulged pyrido-[3,4-d]pyridazine amine derivatives acting as NLRP3 inflammasome inhibitors reported to be useful for the treatment of cancer, metabolic, autoimmune disease, liver, renal, respiratory, cardiovascular and inflammatory disorders, among others. Read More
Yaoya Technology (Shanghai) Co. Ltd. has described Wee1-like protein kinase (Wee1) inhibitors reported to be useful for the treatment of cancer. Read More
Huadong Medicine Co. Ltd. recently detailed the discovery and preclinical characterization of a potent and selective small-molecule glucagon-like peptide-1 receptor (GLP-1R) agonist, HDM-1002, under investigation for the treatment of type 2 diabetes and obesity. Read More
Additional early-stage research and drug discovery news in brief, from: Enveric Biosciences, Gilead, Hookipa, Noxopharm, Palatin Technologies, Recludix. Read More
