Biotherapeutics Discovery, Boehringer Ingelheim Pharmaceutical Inc., Ridgefield, CT, USA.
Medicinal Chemistry, Boehringer Ingelheim Pharma GmbH & Co KG, Biberach, Germany.
MAbs. 2022 Jan-Dec;14(1):2073632. doi: 10.1080/19420862.2022.2073632.
Biotherapeutic optimization, whether to improve general properties or to engineer specific attributes, is a time-consuming process with uncertain outcomes. Conversely, Consensus Protein Design has been shown to be a viable approach to enhance protein stability while retaining function. In adapting this method for a more limited number of protein sequences, we studied 21 consensus single-point variants from eight publicly available CD3 binding sequences with high similarity but diverse biophysical and pharmacological properties. All single-point consensus variants retained CD3 binding and performed similarly in cell-based functional assays. Using Ridge regression analysis, we identified the variants and sequence positions with overall beneficial effects on developability attributes of the CD3 binders. A second round of sequence generation that combined these substitutions into a single molecule yielded a unique CD3 binder with globally optimized developability attributes. In this first application to therapeutic antibodies, adapted Consensus Protein Design was found to be highly beneficial within lead optimization, conserving resources and minimizing iterations. Future implementations of this general strategy may help accelerate drug discovery and improve success rates in bringing novel biotherapeutics to market.
生物治疗优化,无论是改善一般性质还是设计特定属性,都是一个耗时且结果不确定的过程。相反,共识蛋白设计已被证明是一种可行的方法,可以在保留功能的同时提高蛋白质稳定性。在将这种方法应用于数量有限的蛋白质序列时,我们研究了来自八个公开可用的 CD3 结合序列的 21 个共识单点变体,这些序列具有高度相似性,但具有不同的物理化学和药理学性质。所有单点共识变体均保留了 CD3 结合,并在基于细胞的功能测定中表现相似。使用 Ridge 回归分析,我们确定了对 CD3 结合物的可开发性属性具有整体有益影响的变体和序列位置。第二轮序列生成将这些取代组合成一个分子,得到了一个具有全局优化可开发性属性的独特 CD3 结合物。这是首次将适应性共识蛋白设计应用于治疗性抗体,在先导优化中发现它非常有益,节省了资源并减少了迭代次数。未来实施这种通用策略可能有助于加速药物发现并提高将新型生物治疗药物推向市场的成功率。