New Approach Methodologies (NAMs) for drug development are transforming biomedical research by replacing or complementing animal models. More than 90% of experimental compounds fail in clinical trials, underscoring the need for strategies that better capture human biology. Many of these techniques were showcased at the 2026 American Association for Cancer Research (AACR) annual meeting.
Cymirafen is a novel antibody-drug conjugate (ADC) from the University of California that targets leucine-rich repeat-containing G-protein coupled receptor 4 (LGR4)/LGR5/LGR6 and is composed of a potent cytotoxic payload, monomethyl auristatin E (MMAE), plus an Fc domain fused to the receptor binding domain of RSPO1.
Researchers from Cogent Biosciences Inc. presented the preclinical profile of CGT-1263, a pan-KRAS-directed compound that binds both ON and OFF KRAS conformations without affecting HRAS or NRAS.
Researchers from Elpiscience Biopharmaceuticals Inc. hypothesized that dual targeting of the TL1A/DR3 and IL-23 signaling pathways with a single bispecific antibody would exert superior efficacy by reshaping the immune landscape in disorders such as inflammatory bowel disease (IBD). The company has developed a bispecific antibody targeting both TL1A/DR3 and IL-23p19 – ES-302 – for the treatment of IBD.
Klebsiella pneumoniae and Acinetobacter baumannii are both major contributors to the global antimicrobial resistance crisis, causing rapidly progressive and often severe infections. Researchers from Vaxdyn SL and collaborators presented efficacy data for K-Vax, an inactivated, LPS-null whole-cell A. baumannii-based vaccine engineered to display conserved K. pneumoniae outer-membrane proteins on the bacterial cell surface.
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.
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.
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