Rossotti Martin A, González-Techera Andrés, Guarnaschelli Julio, Yim Lucia, Camacho Ximena, Fernández Marcelo, Cabral Pablo, Leizagoyen Carmen, Chabalgoity José A, González-Sapienza Gualberto
a Cátedra de Inmunología; DEPBIO; Facultad de Química; Instituto de Higiene; Universidad de la República ; Montevideo , Uruguay.
MAbs. 2015;7(5):820-8. doi: 10.1080/19420862.2015.1068491.
Recombinant single domain antibodies (nanobodies) constitute an attractive alternative for the production of neutralizing therapeutic agents. Their small size warrants rapid bioavailability and fast penetration to sites of toxin uptake, but also rapid renal clearance, which negatively affects their performance. In this work, we present a new strategy to drastically improve the neutralizing potency of single domain antibodies based on their fusion to a second nanobody specific for the complement receptor CD11b/CD18 (Mac-1). These bispecific antibodies retain a small size (~30 kDa), but acquire effector functions that promote the elimination of the toxin-immunocomplexes. The principle was demonstrated in a mouse model of lethal toxicity with tetanus toxin. Three anti-tetanus toxin nanobodies were selected and characterized in terms of overlapping epitopes and inhibition of toxin binding to neuron gangliosides. Bispecific constructs of the most promising monodomain antibodies were built using anti Mac-1, CD45 and MHC II nanobodies. When co-administered with the toxin, all bispecific antibodies showed higher toxin-neutralizing capacity than the monomeric ones, but only their fusion to the anti-endocytic receptor Mac-1 nanobody allowed the mice to survive a 10-fold lethal dose. In a model of delayed neutralization of the toxin, the anti- Mac-1 bispecific antibodies outperformed a sheep anti-toxin polyclonal IgG that had shown similar neutralization potency in the co-administration experiments. This strategy should have widespread application in the development of nanobody-based neutralizing therapeutics, which can be produced economically and more safely than conventional antisera.
重组单域抗体(纳米抗体)是生产中和治疗剂的一种有吸引力的替代方案。它们的小尺寸保证了快速的生物利用度和快速渗透到毒素摄取部位,但同时也导致快速的肾脏清除,这对它们的性能产生负面影响。在这项工作中,我们提出了一种新策略,通过将单域抗体与针对补体受体CD11b/CD18(Mac-1)的第二个纳米抗体融合,大幅提高其中和效力。这些双特异性抗体保持较小尺寸(约30 kDa),但获得了促进毒素免疫复合物清除的效应功能。该原理在破伤风毒素致死毒性的小鼠模型中得到了验证。选择了三种抗破伤风毒素纳米抗体,并根据重叠表位和毒素与神经节苷脂结合的抑制情况进行了表征。使用抗Mac-1、CD45和MHC II纳米抗体构建了最有前景的单域抗体的双特异性构建体。当与毒素共同给药时,所有双特异性抗体都显示出比单体抗体更高的毒素中和能力,但只有与抗内吞受体Mac-1纳米抗体融合才能使小鼠在10倍致死剂量下存活。在毒素延迟中和模型中,抗Mac-1双特异性抗体的表现优于在共同给药实验中显示出类似中和效力的羊抗毒素多克隆IgG。这种策略应在基于纳米抗体的中和治疗剂的开发中得到广泛应用,这种治疗剂比传统抗血清更经济、更安全。