El Alaoui Meddy, Sivado Eva, Jallas Anne-Catherine, Mebarki Lamia, Dyson Michael R, Perrez Franck, Valsesia-Wittmann Sandrine, El Alaoui Said
Covalab, 1B Rue Jacques Monod, 69500, Bron, France.
Centre Léon Bérard, INSERM 1296 Radiations : défense, Santé et environnement, 28 rue Laennec, 69008, Lyon, France.
Cell Death Discov. 2024 Feb 15;10(1):79. doi: 10.1038/s41420-024-01845-3.
During the last few years Antibody-Drug Conjugates (ADCs) have become one of the most active and very promising therapeutic weapons. Lessons learned from the traditional chemical conjugations (via lysine or cysteine residues of the antibodies) and the clinical studies of the developed ADCs have recently paved the way to the improvement of the conjugation technologies. Use of site-specific conjugation is considered as the promising path for improving the design and development of homogeneous ADCs with controlled Drug-Antibody ratio (DAR). Moreover, some of these conjugations can be applied to antibody fragments such as Fab, scfv and VHH for which random and chemical conjugation showed significant limitations. In this study, we identified a novel small peptide substrate (Q-tag) with high affinity and specificity of bacterial transglutaminase which can be genetically fused to different formats of antibodies of interest for the development of enzymatic site-specific conjugation we named "CovIsolink" platform. We describe the synthesis of chemically defined drugs conjugation in which the site and stoichiometry of conjugation are controlled using a genetically encoded Q-tag peptide with specific amino acids which serves as a substrate of bacterial transglutaminase. This approach has enabled the generation of homogeneous conjugates with DAR 1,7 for full IgG and 0,8 drug ratio for Fab, scfv and VHH antibody fragments without the presence of significant amounts of unconjugated antibody and fragments. As a proof of concept, Q-tagged anti Her-2 (human IgG1 (Trastuzumab) and the corresponding fragments (Fab, scfv and VHH) were engineered and conjugated with different aminated-payloads. The corresponding Cov-ADCs were evaluated in series of in vitro and in vivo assays, demonstrating similar tumor cell killing potency as Trastuzumab emtansine (Kadcyla®) even with lower drug-to-antibody ratio (DAR).
在过去几年中,抗体药物偶联物(ADCs)已成为最活跃且极具前景的治疗武器之一。从传统化学偶联(通过抗体的赖氨酸或半胱氨酸残基)中吸取的经验教训以及已开发的ADCs的临床研究,最近为偶联技术的改进铺平了道路。使用位点特异性偶联被认为是改进具有可控药物-抗体比率(DAR)的均质ADCs设计和开发的有前途的途径。此外,其中一些偶联可应用于抗体片段,如Fab、scfv和VHH,而随机和化学偶联显示出明显的局限性。在本研究中,我们鉴定了一种新型小肽底物(Q标签),其对细菌转谷氨酰胺酶具有高亲和力和特异性,可通过基因融合到感兴趣的不同形式的抗体上,用于开发我们命名为“CovIsolink”平台的酶促位点特异性偶联。我们描述了化学定义的药物偶联物的合成,其中偶联的位点和化学计量通过具有特定氨基酸的基因编码Q标签肽来控制,该肽用作细菌转谷氨酰胺酶的底物。这种方法能够生成DAR为1.7的完整IgG均质偶联物以及DAR为0.8的Fab、scfv和VHH抗体片段的药物比率,且不存在大量未偶联的抗体和片段。作为概念验证,对带有Q标签的抗Her-2(人IgG1(曲妥珠单抗))及其相应片段(Fab、scfv和VHH)进行了工程改造,并与不同的胺化载荷进行偶联。相应的Cov-ADCs在一系列体外和体内试验中进行了评估,结果表明,即使药物-抗体比率(DAR)较低,其肿瘤细胞杀伤效力与曲妥珠单抗恩美曲妥珠单抗(赫赛汀®)相似。