Habermann Jan, Happel Dominic, Bloch Adrian, Shin Charles, Kolmar Harald
Institute for Organic Chemistry and Biochemistry, Technical University of Darmstadt, Darmstadt, Hesse, Germany.
Department of Biomedical Engineering, Johns Hopkins University, Baltimore, Maryland, USA.
Biotechnol J. 2024 Dec;19(12):e202400432. doi: 10.1002/biot.202400432.
The masking of therapeutic antibodies by the installation of a blocking module that can be removed under certain physiological conditions, is becoming increasingly important to improve their safety and toxicity profile. To gain access to such masking units, we used chicken immunization in combination with yeast surface display and a competition-based FACS screening campaign to obtain anti-idiotypic single-chain Fv (scFv) fragments. This approach promotes the identification of functional masking units, since specificity and high affinity do not necessarily guarantee a paratope blocking effect. This strategy was used to isolate a scFv masking unit for the therapeutic antibody 6G11 (BI-1206), which is currently in clinical trials for the treatment of B-cell lymphoma to block the inhibitory Fcγ receptor IIB (CD32b). N-terminal fusion of the anti-idiotypic scFv to the 6G11 light chain successfully abolished binding to FcγRIIB in vitro. For conditional activation, a cleavable linker for the tumor-associated protease MMP-9 was implemented. To improve demasking efficiency, the affinity of the scFv mask was attenuated through rational design. The substitution of one key amino acid in the masking scFv reduced the affinity toward the 6G11 paratope by factor 10 but still mediated 9800-fold blocking of receptor binding. Proteolytic demasking allowed full recovery of therapeutic antibody function in vitro, supporting the concept of conditional antibody activation using this anti-idiotypic binding module.
通过安装可在特定生理条件下去除的阻断模块来掩盖治疗性抗体,对于改善其安全性和毒性特征变得越来越重要。为了获得此类掩盖单元,我们将鸡免疫与酵母表面展示以及基于竞争的荧光激活细胞分选(FACS)筛选活动相结合,以获得抗独特型单链抗体片段(scFv)。这种方法有助于识别功能性掩盖单元,因为特异性和高亲和力不一定能保证抗原决定簇阻断效果。该策略用于分离治疗性抗体6G11(BI - 1206)的scFv掩盖单元,6G11目前正处于治疗B细胞淋巴瘤的临床试验阶段,用于阻断抑制性Fcγ受体IIB(CD32b)。将抗独特型scFv与6G11轻链进行N端融合,成功在体外消除了与FcγRIIB的结合。为了实现条件激活,引入了一种可被肿瘤相关蛋白酶MMP - 9切割的连接子。为了提高去掩盖效率,通过合理设计减弱了scFv掩盖物的亲和力。掩盖性scFv中一个关键氨基酸的替换使对6G11抗原决定簇的亲和力降低了10倍,但仍介导了9800倍的受体结合阻断。蛋白水解去掩盖使治疗性抗体功能在体外完全恢复,支持了使用这种抗独特型结合模块进行条件性抗体激活的概念。