Adoptive cell therapy represents a major step forward in treating hematological cancers. Among its different approaches, chimeric antigen receptor natural killer (CAR-NK) cells are drawing growing interest.
During the first poster session of the 2025 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics, held in Boston, several presentations highlighted novel strategies that move beyond traditional antibody-drug conjugate (ADC) payloads and targets.
A technology that combines transcriptomic data and AI enables a novel approach to drug discovery based on the state of cells, how they behave and which genes they express. The Drugreflector model, developed by scientists at Cellarity Inc., learns from gene expression profiles and predicts which compounds could induce beneficial changes in that cellular state to develop a treatment.
Solu Therapeutics Inc. has identified heterobifunctional cotinine-containing compounds acting as sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers or sodium channel protein type 9 subunit α (SCN9A; Nav1.7) blockers. They are reported to be useful for the treatment of pain, cough and pruritus.
Jiangsu Hengrui Pharmaceuticals Co. Ltd.; Shanghai Hengrui Pharmaceutical Co. Ltd. have synthesized antibody-drug conjugates (ADCs) comprising a claudin 6 (CLDN6)-targeting antibody linked to cytotoxic drug through linker. They are described as potentially useful for the treatment of ovarian cancer.
Merck KGaA has reported proteolysis targeting chimera (PROTACs) comprising an E3 ubiquitin ligase-binding moiety covalently linked to mitogen-activated protein kinase kinase kinase kinase 1 (MAP4K1; HPK1; MEKKK1)-targeting moiety. They are described as potentially useful for the treatment of cancer.
Nippon Shinyaku Co. Ltd. has disclosed compounds inhibiting binding between r(CUG) repeats in DMPK (DM1) and MBNL protein reported to be useful for the treatment of myotonic dystrophy 1.
Positive allosteric modulators (PAMs) of the muscarinic acetylcholine M4 receptor show potential against schizophrenia because they can enhance endogenous acetylcholine signaling, which in turn may mitigate psychotic symptoms as well as improve attention and working memory in individuals with the disorder. However, the efficacy of a promising PAM, CVL-231, has been disappointing in clinical trials.