A modified version of CRISPR-Cas9 has enabled, for the first time, the efficient integration of a large transgene capable of inactivating entire chromosomes into one of the three copies of chromosome 21 in Down syndrome-derived cells. The goal is to silence the extra copy to limit the gene-dosage imbalance that drives many features of trisomy 21. Researchers at Beth Israel Deaconess Medical Center turned to XIST, the long noncoding RNA responsible for the natural silencing of the X chromosome in females. Using this strategy, they achieved integration efficiencies of 20% to 40% and a partial reduction in the overexpression of chromosome 21 genes. Read More
Recent evidence has pointed toward Werner syndrome helicase (WRN) as an attractive target for the management of microsatellite instability-high (MSI-H) tumors, including colorectal, gastric and endometrial cancer mainly. Read More
Researchers from Macrogenics Inc. reported the preclinical characterization of MGC-028, a next-generation ADAM9-targeting antibody-drug conjugate. Read More
Changchun Genescience Pharmaceuticals Co. Ltd. has reported new parathyroid hormone receptor 1 (PTHR1) agonists potentially useful for the treatment of arthritis, fracture, hyperphosphatemia and more. Read More
Although antibodies to PD-L1 are used in the clinic, their benefit is limited by immune exclusion within the local microenvironment. Objective response rates with anti-PD-L1 monotherapy are low due to the heterogeneity of PD-L1 expression, low tumor mutational burden and the highly immunosuppressive tumor microenvironment (TME) of cholangiocarcinoma (CCA).
Mindrank AI Co. Ltd. and Mindrank Therapeutics (Suzhou) New Drug Research have disclosed new Wee1-like protein kinase (Wee1) and/or tyrosine-protein kinase Yes (YES1) inhibitors potentially useful for the treatment of cancer, inflammation and autoimmune diseases. Read More
A joint Tokyo Denki University and Tokyo University of Science patent details new compounds designed for use in the treatment of dyslipidemia. Read More
Researchers from Sanofi SA have detailed the development of a next-generation HER2-targeting T-cell engager (TCE) to increase selectivity for HER2-low tumor cells, while minimizing its effects on normal tissues expressing physiologic levels of HER2. Read More
Tango Therapeutics Inc. has synthesized substituted piperidine-dione molecular glue degraders comprising E3 ubiquitin ligase-binding moiety and acting as HBS1-like protein (HBS1L; HBS1) degradation inducers for use in the treatment of cancer. Read More
Chinese researchers have published data regarding phosphatidylserine (PS) derivatives acting as neuroprotective compounds for Alzheimer’s disease (AD) therapy. Read More
