How Rare Diseases Shape Drug Manufacturing and Development Innovation (Part 1)

In the evolving landscape of pharmaceutical technology, rare disease drug development is emerging as a focal point for innovation and investment. Speaking with The Pharmaceutical Technology® Group, Dan Williams, PhD, CEO, SynaptixBio, highlights the unique challenges and opportunities in targeting such ultra-rare conditions as TUBB4A-related leukodystrophy, a disorder affecting the myelination of nerves in children.

Williams’ experience spans the full spectrum of drug development, from bench science to clinical trials and corporate operations, providing insight into the complexities of bringing novel therapies to market. “About 80% of rare diseases are genetic and have to do with modifications of genes,” he notes, emphasizing the potential for targeted therapies using advanced technologies. For professionals in drug manufacturing and development, the rare disease sector offers scientific challenges as well as significant incentives, including accelerated regulatory pathways and high market value for successful therapies. This shift is drawing increased attention from investors and companies alike, signaling a broader trend toward precision medicine and the rapid advancement of pharmaceutical technology.

About the speaker

Dan Williams, PhD, CEO, SynaptixBio

Dan Williams, PhD, studied biochemistry and physiology at the University of Dundee, after which he worked his way up to senior scientist—managing a cell research group and preclinical development. Following this, he moved to drug development, focusing on the organization and management of both manufacturing and clinical trials. Williams then moved to Adaptimmune, developing cell therapies. He set up the development groups, project managing preclinical research and clinical trials, before becoming vice-president of Research Operations. From there, he moved to Meatable as chief product officer. Williams co-founded SynaptixBio in 2021.