Insmed recently opened a new R&D facility in Cambridge focused on synthetic rescue for serious diseases. Dr Tom Heightman, SVP of Research at the global biopharmaceutical, explains why the company is focused on UK expansion.
Insmed specialises in striving to deliver innovative therapies to transform the lives of patients facing serious diseases.
Its diverse portfolio includes approved and mid-to-late investigational medicines, plus cutting-edge drug discovery focused on areas of greatest patient need.
With an HQ in New Jersey – and offices and research facilities throughout the U.S., Europe and Japan, Insmed is recognised as one of the sector’s best employers, having spent four consecutive years as the No.1 Science Top Employer.
The news that it has expanded its UK-based R&D presence on Babraham Research Campus, in Cambridge, has come as a welcome boost for the UK’s global status in biotechnology innovation.
Speaking at the launch event – attended by guest of honour The Princess Royal – Dr Heightman said: “We are at a pivotal moment where new insights into the biology and genetics of serious diseases are opening new possibilities to identify and develop impactful treatments for conditions that still have few or no effective options.”
Exciting new chapter
Explaining why Insmed has deepened ties to the city, Dr Heightman adds: “Our work in synthetic rescue was developed in Cambridge, and the opening of this site marks an exciting new chapter in our relationship with this region and across the broader life sciences industry here in the UK.”
He adds: “Insmed’s focus is on serious diseases with unmet needs. Our most advanced programs are in pulmonary and inflammatory conditions.
“Across our four research sites in the US and the UK – New Jersey, New Hampshire, San Diego, and Cambridge – we’re expanding into a range of other therapeutic areas.
“In Cambridge, we have a particular focus on CNS diseases, especially neurodegenerative disorders.
“We’ve been based here since the acquisition of Adrestia in August 2023, and our work was previously focused on building a capability for novel target and drug discovery based in start-up labs on the Babraham Research Campus.
“This new site, a 17,000-sq-ft state of the art facility, builds upon Insmed’s existing commitment to innovation and drug development.
“Our continued choice of Cambridge reflects its recognition of the city’s world-leading position in genetics, genomics, and drug discovery, and reputation as the biotech innovation hub of Europe. This supports Insmed’s access to a highly skilled workforce in the city which brings together experts from across continents in a vibrant research ecosystem.”
How does the synthetic rescue platform work?
Dr Heightman continues: “Our new facility will focus on leveraging Insmed’s innovative synthetic rescue platform for genetic diseases.
“Many diseases are caused by the loss of function of a gene. Some of them can be treated by replacing the broken gene through gene therapy, or the gene product through enzyme replacement.
“However, this isn’t always possible, and synthetic rescue looks to the wider gene network for solutions.
“Our cells function through complex networks of many thousands of genes, and nature has taught us that modulating the function of genes other than the broken one may restore healthy cell function – there are some striking examples of individuals with a disease causing gene variant that don’t develop the disease, because they also have a variant in an additional gene that protects them from the disease process.
“We can think of the analogy of a national power grid – if a local substation fails, this can place high stress on neighbouring parts of the grid which can lead to much wider failures.
“If we dial down the power through the neighbouring substations, we may rebalance the system and prevent malfunction.
“Synthetic rescue functions similarly, by modulating cell pathways distal to the broken gene to restore balance. The challenge is to find these productive relationships between disease causing genes and modifier genes.
“Our scalable platform couples cutting-edge molecular and cell biology techniques with sophisticated analysis of human genetics datasets to potentially deliver powerful new ways of treating a broad range of serious and rare diseases.
“We then apply advanced computational design and wet lab techniques to these novel biological targets to discover drug candidates across a range of modalities and bring these forward into clinical testing.”
What makes Insmed’s R&D unique?
Dr Heightman’s answer is unequivocal. “Our R&D is driven by an unparalleled sense of urgency and focus on the needs of the patients we serve.
“Our Cambridge R&D facility uniquely combines novel target discovery using our pioneering synthetic rescue platform with cutting-edge drug discovery techniques.
“We conduct disease biology, wet lab experiments, cellular experiments and screens to identify novel targets as well as chemical design and synthesis all under one roof.
“To maximise effective knowledge creation and transfer, we’ve placed an emphasis on computational science – computational biology, human genetics analysis, in silico drug design, data science – because we really believe that big data of all kinds is critical to our mission. Insmed’s R&D is also unique due to the close collaboration between R&D specialities – both within the Cambridge site, and with other R&D sites around the world.
“And as our research progresses, project hypotheses are tested and validated in disease models of increasing complexity. As each drug discovery effort advances, we use the prototypical drug candidates to provide further validation before selecting the best agent to enter clinical trials, which is where the ultimate validation is tested.”
How quickly could this research translate into treatment?
Dr Heightman explains: “We’re committed to leveraging cutting-edge technology across all disciplines to enhance quality and to accelerate the discovery of new medicines that deliver the greatest possible impact to patients. We’re excited by the advances across our portfolio and look forward to sharing more as our projects progress towards the clinic.
Vision for the future
“Insmed’s vision is to become a globally recognised leading biotech company that empowers great people to deliver, with a profound sense of urgency and compassion, life-altering therapies to small patient populations experiencing big health problems.
“As we begin to realize that vision, we’ll continue to build an innovative pipeline of products and research programs that focus on making a big difference in areas of unmet need.”
“Here at Insmed, we’re pleased to see investment in life sciences being recognised as a priority in the UK – especially the focus on R&D in both the Life Sciences and Industrial strategies.”
