Targeted protein degradation has yet to notch its first approval. But with more than two dozen agents now in clinical trials, the strategy’s ultimate clinical validation appears to be a matter of time.
Multiple myeloma (MM) is a complex disease with poor prognosis and its clinical management still remains a challenge in the field. Since both BCMA and GPRC5D are overexpressed in MM cells, a therapeutic approach targeting both would be of interest. Qilu Pharmaceutical Co. Ltd. is hence developing a dual BCMA- and GPRC5D-targeting antibody – QLS-4131 – for the treatment of MM.
SMARCA4 and SMARCA2 are essential subunits of the SWI/SNF complex, functioning as ATP-dependent chromatin remodelers that regulate gene expression and maintain cellular homeostasis. Recent research has shown that selectively targeting SMARCA2 is an effective cancer treatment strategy, with several compounds already in early clinical testing.
HER3 overexpression in solid tumors is tied to poor prognosis, concretely in breast and non-small-cell lung cancers, where treatment resistance often occurs upon using targeted therapy, highlighting the need for novel targeted therapies against HER3. Since its expression is much higher in tumor than normal cells, HER3 is a robust candidate for antibody-drug conjugate (ADC) therapy.
“I think we’ve come a long way in understanding the importance of this biology. We know it affects men and women, children and adults,” Paul Mischel told the audience during his plenary talk at the 2025 Annual Meeting of the American Association for Cancer Research (AACR 2025). “It’s very prevalent, it’s very devastating. It creates resistance. And we’ve learned some very fundamental rules about this biology that are driving it.”
α7 nicotinic acetylcholine receptors (α7nAChRs), expressed in neurons and astrocytes at lower levels, have been implicated in neuroinflammatory and neurodegenerative processes associated with Alzheimer’s disease (AD). Evidence suggests that α7nAChRs interact with soluble amyloid-β (Aβ), contributing to reactive astrogliosis and formation of Aβ plaques in AD.
Researchers from Aarhus University and Synuca Therapeutics recently presented a novel drug-like small-molecule inhibitor of sarco/endoplasmic reticulum calcium ATPase (SERCA), SYN-4569, with superior pharmacological properties and evaluated its efficacy in vitro and in vivo. SYN-4569 is an orally available compound with good potency, pharmacokinetics and brain-penetrant properties.
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