Pan Andi, Bailey Charles C, Ou Tianling, Xu Jinge, Aristotelous Tonia, Liu Xin, Hu Baodan, Crynen Gogce, Skamangas Nickolas, Bronkema Naomi, Tran Mai H, Mou Huihui, Zhang Xia, Alpert Michael D, Yin Yiming, Farzan Michael, He Wenhui
Skaggs Graduate School, Scripps Research, La Jolla, CA, USA.
The Center for Integrated Solutions to Infectious Diseases, The Broad Institute of MIT and Harvard, Cambridge, MA, USA.
Nat Biomed Eng. 2024 Dec;8(12):1715-1729. doi: 10.1038/s41551-024-01289-1. Epub 2024 Dec 5.
Human proteins repurposed as biologics for clinical use have been engineered through in vitro techniques that improve the affinity of the biologics for their ligands. However, the techniques do not select against properties, such as protease sensitivity or self-reactivity, that impair the biologics' clinical efficacy. Here we show that the B-cell receptors of primary murine B cells can be engineered to affinity mature in vivo the human CD4 domains of the HIV-1-entry inhibitor CD4 immunoadhesin (CD4-Ig). Specifically, we introduced genes encoding the CD4 domains 1 and 2 (D1D2) of a half-life-enhanced form of CD4-Ig (CD4-Ig-v0) into the heavy-chain loci of murine B cells and adoptively transferred these cells into wild-type mice. After immunization, the B cells proliferated, class switched, affinity matured and produced D1D2-presenting antibodies. Somatic hypermutations in the D1D2-encoding region of the engrafted cells improved the binding affinity of CD4-Ig-v0 for the HIV-1 envelope glycoprotein and the inhibitor's ability to neutralize a panel of HIV-1 isolates without impairing its pharmacokinetic properties. In vivo affinity maturation of non-antibody protein biologics may guide the development of more effective therapeutics.
被重新用作临床生物制剂的人类蛋白质是通过体外技术改造而成的,这些技术提高了生物制剂对其配体的亲和力。然而,这些技术并未筛选出诸如蛋白酶敏感性或自身反应性等会损害生物制剂临床疗效的特性。在此,我们表明,原代小鼠B细胞的B细胞受体可经改造,使其在体内对HIV-1进入抑制剂CD4免疫粘附素(CD4-Ig)的人CD4结构域进行亲和力成熟。具体而言,我们将编码半衰期延长形式的CD4-Ig(CD4-Ig-v0)的CD4结构域1和2(D1D2)的基因导入小鼠B细胞的重链基因座,并将这些细胞过继转移到野生型小鼠体内。免疫后,B细胞增殖、类别转换、亲和力成熟并产生呈递D1D2的抗体。植入细胞的D1D2编码区的体细胞超突变提高了CD4-Ig-v0对HIV-1包膜糖蛋白的结合亲和力以及该抑制剂中和一组HIV-1分离株的能力,同时不损害其药代动力学特性。非抗体蛋白生物制剂的体内亲和力成熟可能会指导更有效疗法的开发。