A review article published in Nature Reviews Drug Discovery discusses the current state of N-of-1 therapies. These therapies focus on individuals with rare genetic variants, with treatments developed specifically for them. The article provides a comprehensive roadmap for the development and implementation of these personalized therapies.

Roadmap for N-of-1 therapy development (Jonker et al., 2024)

A new approach in medicine
N-of-1 therapies leverage advanced technologies such as antisense oligonucleotides and gene therapy. These precision treatments offer new hope to patients with conditions that were previously untreatable.

Complex challenges
Despite their promise, N-of-1 therapies face significant challenges. The traditional drug development model does not align with this individualized approach, requiring innovative regulatory and economic frameworks. Without these adaptations, cost and accessibility will remain major barriers.

Hope for rare diseases
With 85% of rare diseases affecting fewer than 1 in 1,000,000 people, N-of-1 therapies represent a crucial step toward personalized medicine. They open the door to treatments fully tailored to individual patients, offering hope to many who previously had none.

The authors emphasize that global collaboration, data sharing, and the development of sustainable models are essential to make these therapies accessible and scalable. The article calls for action to ensure both scientific progress and societal support, so that the promise of N-of-1 therapies can be fully realized.

For more details on the roadmap and further insights into these innovative therapies, please refer to the article in Nature Reviews Drug Discovery.