Molecular Partners AG, Wagistrasse 14, 8952, Schlieren, Switzerland.
Binz Biotech Consulting GmbH, Lüssirainstrasse 52, 6300, Zug, Switzerland.
BioDrugs. 2020 Aug;34(4):423-433. doi: 10.1007/s40259-020-00429-8.
The DARPin drug platform was established with a vision to expand the medical use of biologics beyond what was possible with monoclonal antibodies. It is based on naturally occurring ankyrin repeat domains that are typically building blocks of multifunctional human proteins. The platform allows for the generation of diverse, well-behaved, multifunctional drug candidates. Recent clinical data illustrate the favorable safety profile of the first DARPin molecules tested in patients. With the positive phase III results of the most advanced DARPin drug candidate, abicipar, the DARPin drug platform is potentially about to achieve its first marketing approval. This review highlights some of the key milestones and decisions encountered when transforming the DARPin platform from an academic concept to a biotech drug pipeline engine.
DARPin 药物平台的建立，旨在将生物制剂的医学用途扩展到单克隆抗体所不能达到的范围。它基于天然存在的锚蛋白重复结构域，这些结构域通常是多功能人类蛋白质的构建块。该平台允许生成多样化、表现良好的多功能药物候选物。最近的临床数据说明了在患者中测试的第一批 DARPin 分子的良好安全性概况。随着最先进的 DARPin 药物候选物 abicipar 的 III 期阳性结果，DARPin 药物平台有望首次获得营销批准。本综述强调了将 DARPin 平台从学术概念转化为生物技术药物管道引擎时所遇到的一些关键里程碑和决策。
