Bittner Philipp, Gloger Andreas, Keller Michelle, Oehler Sebastian, Lessing Alice, Zhang Qi, Ma Zhongyao, Su Wenji, Satz Alexander L, Kuai Letian, Zenobi Renato, Scheuermann Jörg
Laboratory of Organic Chemistry, Department of Chemistry and Applied Biosciences, ETH Zurich, Zurich, 8093, Switzerland.
Institute of Pharmaceutical Sciences, Department of Chemistry and Applied Biosciences, ETH Zurich, Zurich, 8093, Switzerland.
Angew Chem Int Ed Engl. 2025 Jul;64(29):e202504470. doi: 10.1002/anie.202504470. Epub 2025 Jun 3.
DNA-encoded Library (DEL) Technology has become a workhorse of drug development, is widely employed in an industrial and academic setting, and an increasing number of drugs developed by DEL technology have entered clinical stage development. While up to billions of compounds can be screened simultaneously in affinity-based selections, the validation and characterization of individual hits discovered from DEL selections is a substantial bottleneck since it can be cumbersome and time-consuming. Here, we describe the use of native mass spectrometry for the speedy hit validation of On-DNA compounds. Through the preservation of noncovalent interactions in the gas phase, complexes of proteins and their respective On-DNA ligands can be analyzed without further need of labeling or immobilization. By utilizing the workflow described in this work, we were able to reliably rank affinities of various purified On-DNA binders or demonstrate binding of On-DNA compounds from unpurified mixtures, mitigating the need for tedious purification steps. Additionally, the methodology described here can offer valuable insight on which moiety of a binding molecule contributes the most to binding, facilitating subsequent medicinal chemistry efforts for lead expansion.
DNA编码文库(DEL)技术已成为药物开发的主力军,广泛应用于工业和学术领域,并且越来越多通过DEL技术开发的药物已进入临床阶段的开发。虽然在基于亲和力的筛选中可以同时筛选多达数十亿种化合物,但从DEL筛选中发现的单个命中化合物的验证和表征却是一个重大瓶颈,因为这可能既繁琐又耗时。在此,我们描述了使用原生质谱对DNA上化合物进行快速命中验证。通过在气相中保留非共价相互作用,可以分析蛋白质与其各自的DNA上配体的复合物,而无需进一步标记或固定。通过利用本工作中描述的工作流程,我们能够可靠地对各种纯化的DNA上结合剂的亲和力进行排名,或证明未纯化混合物中DNA上化合物的结合,从而减少了对繁琐纯化步骤的需求。此外,此处描述的方法可以提供有价值的见解,即结合分子的哪个部分对结合贡献最大,从而有助于后续用于先导物扩展的药物化学研究。