Fabry disease is a rare X-linked lysosomal storage disorder where a deficiency in α-galactosidase A (GLA) results in the pathological accumulation of globotriaosylceramide (Gb3 or GL-3) and other glycosphingolipids in vascular endothelial cells, nerve cells, cardiomyocytes and renal cells.
Huntington’s disease (HD) is caused by the CAG trinucleotide repeat expansion in exon 1 of the huntingtin (HTT) gene, leading to polyglutamine-expanded stretch of mutant huntingtin (mHTT) protein. Previous research has demonstrated that knockdown of HTT could represent an effective strategy for the inhibition of the formation of mHTT protein, and a recent study conducted by researchers from Huidagene Therapeutics Co. Ltd. aimed to assess the potential of high-fidelity Cas12Max (hfCas12Max)-based gene editing therapy as a novel treatment for HD.
At the 30th Annual Congress of the European Society for Gene and Cell Therapy in Brussels this week, researchers presented both preclinical and clinical strategies for applying gene therapy to a functional HIV cure. At a Wednesday session on Infectious Diseases & Vaccines, Alessio Nahmad, of Tabby Therapeutics Ltd., described using B cells edited to express broadly neutralizing antibodies (bnAbs) 3BNC117 to deliver high titers of antibodies in mice.
Opna Bio AG recently provided findings from preclinical studies of novel dual E1A binding protein P300 (EP300) and CREB binding protein (CBP) inhibitors under investigation as potential anticancer agents.
Mucosa-associated lymphoid tissue lymphoma translocation protein 1 (MALT1) is a key regulator of immune signaling that supports proliferation and promotes metastasis while maintaining the tumor microenvironment under immunosuppression. Researchers from Exscientia plc and Evotec SE reported on the characterization of EXS-73565.
Glutaminyl-peptide cyclotransferase-like protein (QPCTL) is a modulator of CD47-SIRPα binding and can be targeted to achieve antitumor responses through myeloid checkpoint blockade. At the recent ESMO meeting in Madrid, researchers from Insilico Medicine Inc. reported the preclinical characterization of ISM-8207, a QPCTL inhibitor with activity against triple-negative breast cancer (TNBC) and diffuse large B-cell lymphoma (DLBCL).
Researchers from Loxo Oncology at Eli Lilly and Co. recently reported the discovery and preclinical evaluation of a new highly potent and selective pan-KRAS inhibitor, LY-4066434, being developed for the treatment of cancer.
Epithelial cell adhesion molecule (EpCAM) is involved in cancer cell invasion while it acts as a negative regulator of adhesion. EpCAM is overexpressed in several cancer types, such as colorectal, ovarian or gastric cancer, and its expression is associated with a worse prognosis.
Artificial intelligence (AI) is in full development, and advances are already used in many fields, including medicine. In oncology, these tools can identify a tumor in an image with 99% accuracy. But they can also miss it if the algorithm was not developed with the right data or the correct decisions. The 2023 European Society for Medical Oncology (ESMO) Congress brought together in Madrid doctors and deep learning experts to discuss the challenges and advances of AI in this area. And what’s better than asking an AI what its trainer has told it about AI?
Data regarding an innovative bispecific antibody – CPL-976 (CPBT-0976) – were recently reported by Celon Pharma SA. Bispecific antibodies targeting more than one antigen on cancer cells improve the specificity and effectiveness of the therapy, and could be utilized for targeting the defense mechanisms of cancer cells, such PD-L1 or EGFR, VEGFR and AXL.
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