Researchers from Xenon Pharmaceuticals Inc. described the preclinical efficacy of XPC-837 in models of Dravet syndrome, a severe developmental and epileptic encephalopathy most commonly caused by de novo loss of function mutations in the SCN1A gene.
Vanda Pharmaceuticals Inc. presented data this week at the annual American Academy of Neurology conference regarding allele-specific antisense oligonucleotides (ASOs) that specifically target the mutant p.A53T allele from the SNCA gene while preserving the expression of the wild-type allele. The mutant allele is associated with increased risk of early-onset Parkinson’s disease (PD) and current ASOs target SNCA regardless of its mutation status.
Despite the success of immunotherapy, it is still limited due to the poor control of which T cells are activated and how strong and how long they are activated. Next-generation T-cell activators should address this limitation by engagement of selective T-cell subsets, allowing stronger control and durable responses. Ipsen SA has presented data regarding their T-cell activator IPN-01203, which is bispecific and selective for Vβ6 and Vβ10 (Vβ6/10) TCR-expressing T cells, alongside with IL-15 receptor coactivation.
When a tumor migrates and colonizes another tissue or organ, it can be identified as a metastasis, but its origin is not always clear. Now, a study based on machine learning has identified DNA-methylation patterns that reveal the type of tissue a cancer comes from when the primary tumor cannot be found. This technique could help guide more specific treatments for patients with cancers of unknown primary, who today often receive broad, nontargeted chemotherapy.
Xuanzhu Biopharmaceutical Co. Ltd. reported positive data from a local phase III study of dirozalkib (Xuan Fei Ning), an ALK inhibitor approved in China to treat patients with advanced non-small-cell lung cancer.
A new study tracking the genomic evolution of chronic myeloproliferative neoplasms has shown that progression to myelofibrosis or acute myeloid leukemia is encoded in mutations that occur years before the transformation is clinically evident.
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.
Recent evidence has suggested that secreted L-amino-acid oxidase, also known as interleukin-4-induced protein 1 (IL4I1), is involved in aromatic amino acid metabolism, as a key immunosuppressive enzyme expressed by tumor-associated myeloid cells, thus suppressing T-cell activation and proliferation within the tumor microenvironment.
Syracuse University recently presented a comprehensive preclinical program describing the rational design and optimization of peptide antagonists targeting the GDF15/GFRAL/RET receptor complex to mitigate nausea, emesis, anorexia and wasting associated with chemotherapy-induced stress signaling.
Transient receptor potential melastatin 3 (TRPM3) is a calcium-permeable TRP channel that is highly expressed in somatosensory neurons, including nociceptors of rodents and humans. Its activation triggers acute pain, making drugs that target TRPM3 a potential approach to alleviate pain. Biohaven Ltd.’s BHV-2100 is the only selective TRPM3 antagonist in clinical development. The company recently disclosed the work leading to its discovery and early development, as well as the chemical structure.
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