Last Friday, Anne, Princess Royal from the British royal family, attended the opening ceremony of Insmed’s new R&D facility in Cambridge, UK. The 17,000 square foot facility is dedicated to synthetic rescue of rare diseases. Princess Anne was given a tour of the site by Will Lewis, Insmed CEO, who commented that he was “immensely proud to show The Princess Royal around our research site.”
In an interview with BioProcess Insider, senior vice president of research Tom Heightman stated that Insmed chose the Cambridge site to “access a different ecosystem of thinking” and that he anticipated significant collaboration with the University of Cambridge.
The facility employs 80 scientists and researchers with “room to expand” over time. Heightman said the facility offers a “state-of-the-art microbiology laboratory.” He added that having a multidisciplinary team work as a cohesive unit contributes to a flourishing research environment.
He emphasized that this collaborative approach differentiates Insmed’s research into rare diseases from that of its competitors. He said that with its proximity to the University of Cambridge, Insmed’s facility has access to some of the best disease experts in the world.
The site will emphasize research on the human genetic code explored in two distinct paths: cellular disease models and the mining of human genetic data through both patient and public databases. By “mining natural variations in human genetic data,” Heightman and his team can identify modifier genes that affect the expression (or lack thereof) of genes integral to disease processes.
Synthetic rescue refers to scenarios in which the loss of one of two essential gene leads to cell death, but the loss of both genes together renders the cell healthy. This parallels the idea of synthetic lethality, where the loss of either gene is survivable, but the loss of both is deadly to the cell. Synthetic lethality has been exploited to develop a range of successful drugs; Insmed hopes to use synthetic rescue to achieve similar success.
This push for synthetic rescue follows Insmed’s purchase of Adrestia, which was built to find therapeutic applications in genetic relationships.
Data mining of the human genome – coupled with cell models genetically altered using CRISPR – allows accurate modeling and understanding of disease. When we asked him about potential timelines for significant research breakthroughs, Heightman commented that while Insmed has “no strict timelines,” the team at Cambridge “has already had significant breakthroughs in neurogenerative disease.” He added, “There are over 7,000 rare diseases, and a significant unmet need. Insmed is interested in areas where they can make a real difference.”
“Our work in synthetic rescue was developed in Cambridge, and the opening of this site marks an exciting new chapter in our relationship,” Heightman said at the opening ceremony.