The Role of Genetic Advances and Partnerships in Rare Disease Drug Development (Part 3)

Editor's note: this interview was originally published on BioPharmInternational.com.

Because small biotechnology companies can leverage their agility and focus to drive innovation—where larger pharmaceutical firms may hesitate—small biotechs are playing a pivotal role in advancing treatments for rare diseases, says Dan Williams, PhD, CEO of SynaptixBio, in the third installment of his interview with Pharmaceutical Technology® Group.

According to Williams, whose company specializes in ultra-rare diseases, “small biotechs ... are able to move things forward ... [and] make decisions extremely quickly,” allowing them to respond rapidly to new data and research opportunities. A significant factor in rare disease research is the genetic basis of these conditions. “About 80% of rare diseases are genetic,” Williams notes, highlighting the impact of recent advances in genome sequencing on diagnostics and drug development.

How are genetic research and strategic partnerships propelling rare disease drugs?

Over the past decade, collaborations with academic institutions, such as the Children’s Hospital of Philadelphia, have deepened understanding of genetic components, accelerating the path from discovery to clinical trials, Williams explains. Despite their strengths, small biotechs face funding challenges. While grant opportunities are increasing in both the United States and United Kingdom, securing resources remains a major hurdle, Williams points out.

As rare disease treatments progress through clinical development, partnerships with larger pharmaceutical companies become essential. These collaborations bring the rigorous methodologies and resources needed to bring new therapies to market, says Williams. In addition, patient advocacy groups are also shaping the landscape, providing critical input on treatment priorities and quality of life improvements. As research momentum builds, the integration of genetic insights, agile biotech strategies, and collaborative partnerships is set to transform the future of rare disease therapeutics, he concludes.

Click the video above to view this interview.

Click here for Part 1. Click here for Part 2.

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.