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二硫键约束肽支架可实现稳健的肽类治疗药物发现平台。

Disulfide-constrained peptide scaffolds enable a robust peptide-therapeutic discovery platform.

机构信息

Departments of Biological Chemistry, Genentech, Inc., South San Francisco, California, United States of America.

Department of Peptide Therapeutics, Genentech, Inc., South San Francisco, California, United States of America.

出版信息

PLoS One. 2024 Mar 28;19(3):e0300135. doi: 10.1371/journal.pone.0300135. eCollection 2024.

Abstract

Peptides present an alternative modality to immunoglobulin domains or small molecules for developing therapeutics to either agonize or antagonize cellular pathways associated with diseases. However, peptides often suffer from poor chemical and physical stability, limiting their therapeutic potential. Disulfide-constrained peptides (DCP) are naturally occurring and possess numerous desirable properties, such as high stability, that qualify them as drug-like scaffolds for peptide therapeutics. DCPs contain loop regions protruding from the core of the molecule that are amenable to peptide engineering via direct evolution by use of phage display technology. In this study, we have established a robust platform for the discovery of peptide therapeutics using various DCPs as scaffolds. We created diverse libraries comprising seven different DCP scaffolds, resulting in an overall diversity of 2 x 1011. The effectiveness of this platform for functional hit discovery has been extensively evaluated, demonstrating a hit rate comparable to that of synthetic antibody libraries. By utilizing chemically synthesized and in vitro folded peptides derived from selections of phage displayed DCP libraries, we have successfully generated functional inhibitors targeting the HtrA1 protease. Through affinity maturation strategies, we have transformed initially weak binders against Notch2 with micromolar Kd values to high-affinity ligands in the nanomolar range. This process highlights a viable hit-to-lead progression. Overall, our platform holds significant potential to greatly enhance the discovery of peptide therapeutics.

摘要

肽为开发激动或拮抗与疾病相关的细胞通路的治疗药物提供了一种替代免疫球蛋白结构域或小分子的方法。然而,肽通常稳定性差,限制了其治疗潜力。二硫键约束肽(DCP)是天然存在的,具有许多理想的特性,如高稳定性,使其成为肽治疗药物的类药支架。DCP 包含从分子核心突出的环区,可通过使用噬菌体展示技术进行直接进化来进行肽工程改造。在这项研究中,我们建立了一个使用各种 DCP 作为支架发现肽治疗药物的强大平台。我们创建了包含七个不同 DCP 支架的多样化文库,总体多样性为 2 x 1011。该平台对功能命中发现的有效性进行了广泛评估,显示出与合成抗体文库相当的命中率。通过利用噬菌体展示 DCP 文库选择的化学合成和体外折叠的肽,我们成功地生成了针对 HtrA1 蛋白酶的功能性抑制剂。通过亲和成熟策略,我们将最初对 Notch2 具有微摩尔 Kd 值的弱结合物转化为纳米摩尔范围内的高亲和力配体。这个过程突出了可行的从命中到先导的进展。总的来说,我们的平台具有极大地增强肽治疗药物发现的潜力。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0f0e/10977697/e6a87b0e4e67/pone.0300135.g001.jpg

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