Mayo Foundation for Medical Education Research (MFMER) and the University of Minnesota have disclosed new bromodomain-containing protein 4 (BRD4; HUNK1) inhibitors potentially useful for the treatment of cancer, inflammatory and cardiovascular disorders.
Entos Pharmaceuticals Inc. has established a collaboration with the L-CMD Research Foundation with the aim of developing a curative therapy for LMNA-related congenital muscular dystrophy (L-CMD).
Selective inhibition of Werner syndrome helicase (WRN) has been shown to trigger extensive DNA damage and cell death specifically in microsatellite instability-high (MSI-H) cancers, highlighting WRN as a tumor-selective target with potential for precision oncology approaches beyond immunotherapy. Researchers from Eikon Therapeutics Inc. presented the preclinical profile of EIK-1005, a WRN inhibitor.
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.
Addex Therapeutics Ltd. has announced findings from the evaluation of its γ-aminobutyric acid subtype B receptor (GABA[B]) positive allosteric modulator (PAM) in a nonhuman primate (NHP) model of chronic cough. In the study, the GABA(B) PAM drug candidate significantly reduced citric acid-induced cough frequency.
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.
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.
RSS
