<p>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.</p> Graphical Abstract <p></p>

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The translational journey of cancer nanomedicines: biological and entrepreneurial lessons learned

  • Cristianne Johanna Ferdinand Rijcken

摘要

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.

Graphical Abstract