Rijcken Cristianne Johanna Ferdinand
Cristal Therapeutics, Oxfordlaan 55, Maastricht, 6229EV, The Netherlands.
Liberates, Havenstraat 4c04, Maastricht, 6211GJ, The Netherlands.
Drug Deliv Transl Res. 2025 Apr 30. doi: 10.1007/s13346-025-01867-z.
Despite exhaustive investments, the breakthrough potential of nanomedicines (NM) is not yet realized. Whilst Doxil and covid-19 vaccines demonstrated certain benefits, many NM failed in clinical development. Lies the true reason for this limited success in inappropriate assumptions, incorrect approaches, or other omissions? This note describes the translational journey of CPC634 (docetaxel entrapping core-crosslinked polymeric micelles) and illustrates lessons learned in drug product development. Scientific elements are to understand the pathophysiology of the diseased tissue, the journey of NM upon administration and resulting drug release and the induced pharmacodynamic effects over time, particularly in actual patients. Industrial elements comprise market-product fit, target product profile, competitive benchmarking, while development efficiency focuses to generate a positive business case. A goal-oriented product design which is validated by external experts increases chances of development success and assures investor-readiness. NM development will progress by aligning fundamental biological insights with industrial product requirements, driving therapeutic breakthroughs.
尽管进行了大量投资，但纳米药物（NM）的突破潜力尚未实现。虽然多西紫杉醇脂质体（Doxil）和新冠疫苗显示出一定益处，但许多纳米药物在临床开发中失败了。这种有限成功的真正原因是不恰当的假设、错误的方法或其他疏忽吗？本报告描述了CPC634（载有多西他赛的核心交联聚合物胶束）的转化过程，并阐述了在药品开发中吸取的经验教训。科学要素包括了解患病组织的病理生理学、纳米药物给药后的历程以及由此产生的药物释放和随时间推移所诱导的药效学效应，特别是在实际患者中。产业要素包括市场与产品匹配度、目标产品概况、竞争基准，而开发效率则侧重于产生积极的商业案例。由外部专家验证的目标导向型产品设计可增加开发成功的机会，并确保达到投资者的要求。通过将基础生物学见解与产业产品要求相结合，纳米药物开发将取得进展，推动治疗突破。
