Genome-wide association studies (GWAS) have identified multiple loci associated with complex diseases, but these are mostly on regulatory genes in the non-coding part of the genome and it has proved difficult to identify the effector genes that they control. Now, researchers in the U.K. have shown how single cell sequencing at scale can be used to precisely link non-coding GWAS loci to specific protein coding genes and cell types.
Simcere Zaiming Pharmaceutical Co. Ltd. has divulged new mitogen-activated protein kinase (MAPK) inhibitors found to be potentially useful for the treatment of cancer.
New York University has discovered new analogues of mannose-6-phosphate reported to be potentially useful for the treatment of memory impairment, neurodegeneration and neurodevelopmental disorders, among others.
Researchers from Jiangsu Hengrui Pharmaceuticals Co. Ltd. and Shanghai Hengrui Pharmaceutical Co. Ltd. have reported molecular glue degraders acting as proto-oncogene Vav (VAV1) degradation inducers found to be potentially useful for the treatment of cancer, inflammatory disorders and autoimmune diseases.
Artan Biotechnologies LLC has completed a $200,000 seed raise to fund preclinical advancement of the company’s proprietary engineered suppressor platform, which targets nonsense mutations.
Previous work found that certain short RNAs can induce cell death in a RISC-dependent fashion by targeting several networks of survival genes simultaneously, therefore triggering multiple cell death pathways. This form of cell death was named death induced by survival gene elimination, or DISE, an effect that depends on a toxic 6-mer seed.
About 90% of brain metastases are often limited therapeutically speaking due to the impermeable blood-brain barrier (BBB). Nanocarry Therapeutics Ltd. has presented AxS007, a novel insulin-mediated nanocarrier that delivers multiple copies of trastuzumab and pertuzumab across the BBB, using native insulin as a brain transporter and increasing brain exposure.
The University of Texas MD Anderson Cancer Center reported findings from studies of CBT-001-2334, a radionuclide peptide targeting carbonic anhydrase IX (CAIX/CA9) designed for diagnostic gallium labeling and downstream therapeutic isotope pairing. It features a DOTA chelator for use in theranostic applications through chelating either diagnostic or therapeutic radionuclides. Because it has limited expression in normal tissue, CAIX is an attractive target in clear cell renal cell carcinoma (ccRCC).