Immunology Therapeutic Area UCB Pharma Slough UK.
Research and Development Prisys Biotechnologies Pudong China.
Animal Model Exp Med. 2021 Nov 19;4(4):359-368. doi: 10.1002/ame2.12185. eCollection 2021 Dec.
Chronic kidney disease (CKD) has a high global prevalence and large unmet need. Central to developing new CKD therapies are in vivo models in CKD. However, next-generation antibody, protein, and gene therapies are highly specific, meaning some do not cross-react with rodent targets. This complicates preclinical development, as established in vivo rodent models cannot be utilized unless tool therapeutics are also developed. Tool compounds can be difficult to develop and, if available, typically have different epitopes, sequences, and/or altered affinity, making it unclear how efficacious the lead therapeutic may be, or what dosing regimen to investigate. To address this, we aimed to develop a nonhuman primate model of CKD.
In vivo rodent unilateral ureteral obstruction (UUO) models kidney fibrosis and is commonly used due to its rapidity, consistency, and ease. We describe translation of this model to the cynomolgus monkey, specifically optimizing the model duration to allow adequate time for assessment of novel therapeutics prior to the fibrotic plateau.
We demonstrated that disease developed more slowly in cynomolgus monkeys than in rodents post-UUO, with advanced fibrosis developing by 6 weeks. The tubulointerstitial fibrosis in cynomolgus monkeys was more consistent with human obstructive disease than in rodents, having a more aggressive tubular basement expansion and a higher fibroblast infiltration. The fibrosis was also associated with increased transglutaminase activity, consistent with that seen in patients with CKD.
This cynomolgus monkey UUO model can be used to test potential human-specific therapeutics in kidney fibrosis.
慢性肾脏病(CKD)在全球的发病率很高,且存在大量未得到满足的医疗需求。开发新的 CKD 疗法的关键在于 CKD 的体内模型。然而,下一代抗体、蛋白质和基因疗法具有高度特异性,这意味着它们不会与啮齿动物的靶标发生交叉反应。这使得临床前开发变得复杂,因为除非开发出工具治疗药物,否则无法使用现有的体内啮齿动物模型。工具化合物可能难以开发,而且如果有,通常具有不同的表位、序列和/或改变的亲和力,这使得不清楚先导治疗药物的疗效如何,或者应该研究哪种给药方案。为了解决这个问题,我们旨在开发一种 CKD 的非人类灵长类动物模型。
体内啮齿动物单侧输尿管梗阻(UUO)模型会导致肾脏纤维化,由于其快速、一致和易于操作而被广泛应用。我们描述了该模型在食蟹猴中的转化,特别是优化了模型持续时间,以便在纤维化平台之前有足够的时间评估新的治疗药物。
我们证明,UUO 后食蟹猴的疾病发展比啮齿动物缓慢,在 6 周后出现晚期纤维化。食蟹猴的肾小管间质纤维化比啮齿动物更符合人类梗阻性疾病,具有更具侵袭性的肾小管基底膜扩张和更高的成纤维细胞浸润。纤维化还与转谷氨酰胺酶活性增加有关,这与 CKD 患者的情况一致。
这种食蟹猴 UUO 模型可用于测试潜在的人类特异性治疗药物在肾脏纤维化中的作用。