Repeat expansions of two or more base pairs cause dozens of neurological disorders – Huntington’s disease, which is caused by an expansion of the triplet CAG in the coding sequence for huntingtin, is perhaps the most famous one. Now, investigators at Stanford University have shown that cancer genomes, too, frequently feature repeat expansions. Read More
The farnesoid X receptor (FXR), a bile acid receptor, plays a direct role regulating innate immune cells in the gut, and treating mice with an FXR agonist improved symptoms of inflammatory bowel disease (IBD). The findings provide a link between diet and innate immunity, and could lead to better ways to treat IBD. “The intestine is a major target for inflammation and inflammatory disease, particularly in the modern Western culture, where high-fat diets are becoming very prevalent,” Ronald Evans told BioWorld. Read More
Seelos Therapeutics Inc. has announced in vivo data demonstrating that a single dose of SLS-004 downregulated the production of α-synuclein in an established α-synuclein overexpressing animal model of Parkinson's disease. Read More
SLN-140 (Silence Therapeutics plc) is a novel small interfering RNA (siRNA) targeting protein S for the treatment of hemophilia A (HA). Researchers from the University of Bern recently presented data from studies conducted in animal models of HA, performed to evaluate the safety and efficacy of SLN-140. Read More
Halda Therapeutics Opco Inc. has patented proteolysis-targeting chimeric (PROTAC) compounds comprising an E3 ubiquitin ligase cereblon (CRBN)-binding moiety covalently bonded to a protein-targeting moiety via linker reported to be useful for the treatment of cancer and hepatitis. Read More
TSC22 domain family member 3 (TSC22D3) is a glucocorticoid-induced gene that plays a key regulatory role in immunosuppression and cell proliferation. Its prognostic usefulness in acute myeloid leukemia (AML) has not been deeply investigated yet. Read More
Genprex Inc. has entered into a license agreement with the University of Pittsburgh designed to strengthen its diabetes program. The agreement grants Genprex a worldwide, exclusive license to certain patent applications and related technology and a worldwide, nonexclusive license to use certain related know-how, all related to modulating autoimmunity in type 1 diabetes by using gene therapy. Read More
Innocure Therapeutics Inc. has divulged piperidinediones as proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase cereblon (CRBN)-binding moiety covalently linked to bromodomain-containing protein 4 (BRD4)- and/or tyrosine-protein phosphatase non-receptor type 11 (PTPN11)-targeting moiety via linker reported to be useful for the treatment of cancer. Read More
Several STING agonists have demonstrated antitumor efficacy in preclinical studies and are currently under clinical development. However, systemic administration of STING agonists may have adverse effects, while intratumoral injection is limited by tumor accessibility. Therefore, systemic delivery of STING agonists specifically targeted to tumors emerges as a potential strategy to overcome these limitations. Read More
Novartis AG has identified triazolo-pyrimidine analogues acting as Werner syndrome ATP-dependent helicase (WRN) inhibitors reported to be useful for the treatment of cancer. Read More
Researchers from Fudan University presented data from a study that aimed to assess the significance of a newly found long noncoding RNA (lncRNA), Ewing sarcoma-associated transcript 1 (EWSAT1), in hepatocellular carcinoma (HCC) metastasis. Read More
