Cullen Eye Institute, Department of Ophthalmology, Baylor College of Medicine, Houston, TX 77030, USA.
Everglades Biopharma, LLC, Houston, TX 77098, USA.
Int J Mol Sci. 2024 Sep 1;25(17):9507. doi: 10.3390/ijms25179507.
Secretogranin III (Scg3) is a diabetic retinopathy (DR)-restricted angiogenic factor identified in preclinical studies as a target for DR therapy. Previously, our group generated and characterized ML49.3, an anti-Scg3 monoclonal antibody (mAb) which we then converted into an EBP2 humanized antibody Fab fragment (hFab) with potential for clinical application. We also generated anti-Scg3 mT4 mAb and related EBP3 hFab. In this study, to identify the preferred hFab for DR therapy, we compared all four antibodies for binding, neutralizing and therapeutic activities in vitro and in vivo. Octet binding kinetics analyses revealed that ML49.3 mAb, EBP2 hFab, mT4 mAb and EBP3 hFab have Scg3-binding affinities of 35, 8.7, 0.859 and 0.116 nM, respectively. Both anti-Scg3 EBP2 and EBP3 hFabs significantly inhibited Scg3-induced proliferation and migration of human umbilical vein endothelial cells in vitro, and alleviated DR vascular leakage and choroidal neovascularization with high efficacy. Paired assays in DR mice revealed that intravitreally injected EBP3 hFab is 26.4% and 10.3% more effective than EBP2 hFab and aflibercept, respectively, for ameliorating DR leakage. In conclusion, this study confirms the markedly improved binding affinities of hFabs compared to mAbs and further identifies EBP3 hFab as the preferred antibody to develop for anti-Scg3 therapy.
分泌颗粒蛋白 III(Scg3)是一种糖尿病视网膜病变(DR)特异性血管生成因子,在临床前研究中被确定为 DR 治疗的靶点。此前,我们的团队生成并表征了 ML49.3,这是一种抗 Scg3 单克隆抗体(mAb),随后我们将其转化为具有临床应用潜力的 EBP2 人源化抗体 Fab 片段(hFab)。我们还生成了抗 Scg3 mT4 mAb 和相关的 EBP3 hFab。在这项研究中,为了确定用于 DR 治疗的首选 hFab,我们比较了这四种抗体在体外和体内的结合、中和和治疗活性。Octet 结合动力学分析显示,ML49.3 mAb、EBP2 hFab、mT4 mAb 和 EBP3 hFab 与 Scg3 的结合亲和力分别为 35、8.7、0.859 和 0.116 nM。两种抗 Scg3 的 EBP2 和 EBP3 hFabs 均显著抑制 Scg3 诱导的人脐静脉内皮细胞增殖和迁移,并且高效缓解 DR 血管渗漏和脉络膜新生血管形成。DR 小鼠的配对检测显示,玻璃体内注射的 EBP3 hFab 分别比 EBP2 hFab 和 aflibercept 更有效地改善 DR 渗漏,效力提高了 26.4%和 10.3%。综上所述,这项研究证实了 hFab 与 mAb 相比具有明显提高的结合亲和力,并进一步确定 EBP3 hFab 是开发抗 Scg3 治疗的首选抗体。