CTNNB1 syndrome is a rare neurodevelopmental disorder that is caused by mutations in the gene encoding β-catenin, CTNNB1, which plays a critical role in neuronal development, synapse formation and brain maturation.
About 50% of patients with small intestine neuroendocrine tumors (SI-NETs) have their prognosis negatively impacted by the development of mesenteric fibrosis, but the mechanisms behind these are poorly understood. In work at Sun Yat-sen University, single-cell RNA sequencing was performed on five primary tumor specimens and their corresponding adjacent nontumoral tissues.
Researchers from Iomx Therapeutics AG presented the discovery and preclinical evaluation of IOMX-0675, a novel LILRB1 and LILRB2 cross-specific antibody being developed for the treatment of solid tumors.
α1-Antitrypsin deficiency is a hereditary disorder that affects the liver in children and adults, as well as the lungs in adults. The disease is mostly caused by the Z allele mutation in the SERPINA1 gene, where a glutamic acid amino acid is substituted by lysine (E342K) leading to protein misfolding and aggregation.
Many patients with peripheral nerve diseases do not have a sufficient regenerative response because of genetic inheritance, infections or chronic disease, leading them to lifelong pain and disability.
Researchers from Poseida Therapeutics Inc. presented preclinical data for P-FVIII-101, a novel nonviral gene therapy being developed for the treatment of hemophilia A.
Genmab A/S, in collaboration with Biontech SE, has presented data on a novel OX40 agonist antibody –Hexabody-OX40 (GEN-1055/BNT-315), developed using Genmab’s proprietary Hexabody platform, which promotes the formation of antibody hexamers upon target binding to cell surfaces.
Lowering intraocular pressure (IOP) is still the only approach for treating glaucoma, in which there is mitochondrial damage in the retinal ganglion cells (RGCs). Activating survival pathways in RGCs was thus hypothesized by Indiana University scientists as a strategy for treating glaucoma independently of IOP modulation.
Investigators at Poseida Therapeutics Inc. developed P-KLKB1-101, a nonviral KLKB1 gene editing therapy, being developed for the treatment of hereditary angioedema (HAE).
Researchers from Deutsches Krebsforschungszentrum DKFZ and affiliated organizations presented data for the novel inhibitor of DNA methyltransferase (DNMT) and histone methyltransferase G9a (HMT-G9a), CM-272, being developed for the treatment of acute myeloid leukemia (AML).
RSS
