The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap.

In recent years, a small number of people with rare diseases caused by unique genetic variants have been treated with therapies developed specifically for them. This pioneering field of genetic N-of-1 therapies is evolving rapidly, giving hope for the individualized treatment of people living with very rare diseases. In this Review, we outline the concept of N-of-1 individualized therapies, focusing on genetic therapies, and illustrate advances and challenges in the field using cases for which therapies have been successfully developed.

Challenges With the Development of Biosimilars in Asia for Western Markets: An Overview and Suggested Solutions.

There is a great interest from companies located in Asia to bring biosimilars into Western markets such as the United States, European Union, and Canada, because these are lucrative markets with patents expiring or close to expiry for many originator biologics. Although many sponsors are successfully developing and marketing biosimilars for their own countries, there is an increasing interest among companies in China, Hong Kong, India, Korea, Taiwan, and other Asian countries in targeting markets in the West. As a result, there is widespread interest among these companies in learning about the requirements for regulatory approval in Western countries.