Biotherapeutics Discovery, Boehringer Ingelheim Pharmaceuticals Inc, Ridgefield, Connecticut, USA.
Biotherapeutics Discovery, Boehringer Ingelheim Pharmaceuticals Inc, Ridgefield, Connecticut, USA.
J Biol Chem. 2024 Jan;300(1):105555. doi: 10.1016/j.jbc.2023.105555. Epub 2023 Dec 10.
Discovery and optimization of a biotherapeutic monoclonal antibody requires a careful balance of target engagement and physicochemical developability properties. To take full advantage of the sequence diversity provided by different antibody discovery platforms, a rapid and reliable process for humanization of antibodies from nonhuman sources is required. Canonically, maximizing homology of the human variable region (V-region) to the original germline was believed to result in preservation of binding, often without much consideration for inherent molecular properties. We expand on this approach by grafting the complementary determining regions (CDRs) of a mouse anti-LAG3 antibody into an extensive matrix of human variable heavy chain (VH) and variable light chain (VL) framework regions with substantially broader sequence homology to assess the impact on complementary determining region-framework compatibility through progressive evaluation of expression, affinity, biophysical developability, and function. Specific VH and VL framework sequences were associated with major expression and purification phenotypes. Greater VL sequence conservation was correlated with retained or improved affinity. Analysis of grafts that bound the target demonstrated that initial developability criteria were significantly impacted by VH, but not VL. In contrast, cell binding and functional characteristics were significantly impacted by VL, but not VH. Principal component analysis of all factors identified multiple grafts that exhibited more favorable antibody properties, notably with nonoptimal sequence conservation. Overall, this study demonstrates that modern throughput systems enable a more thorough, customizable, and systematic analysis of graft-framework combinations, resulting in humanized antibodies with improved global properties that may progress through development more quickly and with a greater probability of success.
发现和优化生物治疗性单克隆抗体需要在靶标结合和物理化学可开发性之间进行仔细平衡。为了充分利用不同抗体发现平台提供的序列多样性,需要一种快速可靠的方法来对非人类来源的抗体进行人源化。传统上,最大限度地提高人类可变区(V 区)与原始种系的同源性被认为可以保持结合,而通常很少考虑固有分子特性。我们通过将小鼠抗 LAG3 抗体的互补决定区(CDR)移植到广泛的人类重链(VH)和轻链(VL)框架区矩阵中,进一步扩展了这种方法,该矩阵与原始种系具有更大的序列同源性,以评估通过渐进评估表达、亲和力、生物物理可开发性和功能对互补决定区-框架兼容性的影响。特定的 VH 和 VL 框架序列与主要的表达和纯化表型相关。VL 序列的更大保守性与保留或提高的亲和力相关。对结合靶标的移植体进行分析表明,最初的可开发性标准受到 VH 的显著影响,但不受 VL 的影响。相比之下,细胞结合和功能特征受到 VL 的显著影响,但不受 VH 的影响。对所有因素的主成分分析确定了多个表现出更好抗体特性的移植体,尤其是在非最佳序列保守性方面。总体而言,这项研究表明,现代高通量系统能够更全面、可定制和系统地分析移植体-框架组合,从而产生具有改善的整体特性的人源化抗体,这些抗体可能更快地通过开发阶段,并且成功的可能性更大。