Pharmaceutical Discovery and Preclinical Development1

Happy New Year!
In the search for individualized medicine, researchers are exploring the emerging field of genetic N-of-1 therapies. These personalized treatments are designed for individuals with rare diseases caused by unique genetic mutations, including ultra-rare conditions. However, this approach differs significantly from traditional clinical trial principles.
A recent review highlights the rapid advancement of N-of-1 therapies, showcasing successful cases where tailored genetic interventions have transformed lives. The authors stress the need for new frameworks to address the regulatory, ethical, and practical challenges unique to this field.
Please let me know if you experience difficulties in accessing the article.
Jonker, A.H., Tataru, EA., Graessner, H. et al. The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap. Nat Rev Drug Discov 24, 40–56 (2025).
https://doi.org/10.1038/s41573-024-01059-3 
Best regards. 

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Sudip Das, MPharm, PhD
Distinguished Faculty, Butler University
Professor of Pharmaceutics & Drug Delivery
4600 Sunset Avenue, Indianapolis, IN 46208-3485
E-mail: [email protected]
Webpage: https://research.butler.edu/nanomedicine/
LinkedIn: http://www.linkedin.com/in/sudipkdas
Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
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Sudip,
This is one of the articles that help to expand my knowledge. While in the middle of our academic work, we tend to forget to expand the alternate development models while sticking to what we know. We want individualized therapies, but this paper has expanded my knowledge base. Appreciate you taking time to highlight this article.

Thank you.

Bhaskar


Original Message:
Sent: 1/6/2025 2:54:00 PM
From: Sudip Das
Subject: The Future of Personalized Medicine: The N-of-1 Therapies

Happy New Year!
In the search for individualized medicine, researchers are exploring the emerging field of genetic N-of-1 therapies. These personalized treatments are designed for individuals with rare diseases caused by unique genetic mutations, including ultra-rare conditions. However, this approach differs significantly from traditional clinical trial principles.
A recent review highlights the rapid advancement of N-of-1 therapies, showcasing successful cases where tailored genetic interventions have transformed lives. The authors stress the need for new frameworks to address the regulatory, ethical, and practical challenges unique to this field.
Please let me know if you experience difficulties in accessing the article.
Jonker, A.H., Tataru, EA., Graessner, H. et al. The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap. Nat Rev Drug Discov 24, 40–56 (2025).
https://doi.org/10.1038/s41573-024-01059-3 
Best regards. 

------------------------------
Sudip Das, MPharm, PhD
Distinguished Faculty, Butler University
Professor of Pharmaceutics & Drug Delivery
4600 Sunset Avenue, Indianapolis, IN 46208-3485
E-mail: [email protected]
Webpage: https://research.butler.edu/nanomedicine/
LinkedIn: http://www.linkedin.com/in/sudipkdas
Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
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