Insmed Opens Innovation Centre, Pioneers Synthetic Rescue Therapies

Insmed, a US-based biopharmaceutical company, has officially inaugurated its new innovation centre located within the Babraham Research Campus in Cambridge. The opening ceremony, held in mid-July 2023, was graced by Her Royal Highness, The Princess Royal. This state-of-the-art facility spans 17,000 square feet and marks a significant strategic shift for Insmed as it ventures into the realm of synthetic rescue—a genetic approach designed to repair cellular pathways to restore normal function.

At the forefront of this initiative is Dr Tom Heightman, Senior Vice President and Head of Research at the Cambridge site. With over 25 years in the pharmaceutical industry, Heightman has previously held notable positions at companies including GlaxoSmithKline and AstraZeneca. He led the transformation of Adrestia prior to its acquisition by Insmed in 2023, forming the core of Insmed’s new UK research and development hub.

“When Insmed approached us for a potential acquisition, we became very interested in their science, success, and culture. We saw it was a great fit between the two companies,” Heightman stated. The integration of Adrestia’s early-stage target discovery and drug discovery platform aligns with Insmed’s vision of establishing a long-term therapeutic pipeline.

Understanding Synthetic Rescue

Synthetic rescue is emerging as a promising alternative to synthetic lethality, a concept prevalent in oncology. In synthetic lethality, two concurrent gene disruptions lead to cell death; conversely, synthetic rescue aims to find alternative pathways that can restore health when one essential gene is compromised. Insmed’s platform employs a combination of whole-genome CRISPR screens, high-throughput validation, and extensive analysis of human genetic datasets, coupled with computational drug design and chemistry.

“What makes this facility unique is two-fold,” Heightman explained. “First, the synthetic rescue platform itself. Second, we can execute end-to-end drug discovery in one place: genetic discovery, data science, computational chemistry, and wet-lab synthesis.” Although Insmed currently employs around 80 staff, it has established a fully integrated discovery hub that offers a breadth of scientific expertise unusual for a company of its size.

The focus is primarily on rare genetic disorders, particularly those impacting the central nervous system and neurodegenerative diseases. Heightman emphasizes the broad applicability of synthetic rescue, noting that there are nearly 7,000 recognized rare diseases, many lacking a clear mechanistic understanding. This complexity makes traditional drug discovery challenging. To address this, the Cambridge team is developing assays and cellular models from the ground up to gain deeper biological insights and assess patient impacts.

One significant advantage of synthetic rescue is its ability to target compensatory pathways, even when the original genetic mutation cannot be corrected. This opens therapeutic avenues in genetic conditions previously deemed untreatable.

Collaborative Research Environment

Insmed’s Cambridge facility complements its existing research sites in San Diego, New Hampshire, and New Jersey, each specializing in different modalities. San Diego focuses on gene therapy, New Hampshire on biologics, and New Jersey on lipid-formulated small molecules. Heightman describes these sites as “semi-independent hubs,” convening every six months to share breakthroughs and foster cross-pollination of ideas.

Looking ahead, Insmed aims to facilitate the shared use of expensive equipment across its research hubs, minimizing duplication while maximizing resource efficiency. Heightman indicated that the first half of 2025 has already yielded promising breakthroughs across several early-stage projects led by the Cambridge team. The current objective is to generate preclinical data to advance these discoveries toward potential prototype medicines.

“We intend to make the second half of the year scientifically rich—more data and more clarity on molecules progressing through preclinical development,” Heightman noted.

Beyond internal collaboration, Insmed is also promoting dialogues between researchers and clinicians. Heightman pointed out that clinical teams are increasingly reaching out to researchers, creating a valuable exchange of knowledge rather than a one-sided communication flow.

While the role of artificial intelligence (AI) in drug discovery is a hot topic, Heightman remains cautious. “AI works best in areas where we have deep datasets, and we’re seeing success in specific, well-defined problems. The hope is that future generations of AI will integrate different data types more holistically. For now, we still need human scientists to drive the process,” he explained.

Insmed continues to support its existing portfolio, including brensocatib for bronchiectasis, which is being advanced through public market fundraising. In June 2025, the company priced a public offering of $750 million to fund its research and commercialization initiatives, demonstrating strong investor confidence despite previous operating losses.

As Insmed deepens its involvement in the UK life sciences sector, its emphasis on synthetic rescue signals a pivotal shift in approaching genetic diseases. While the science is still in the early stages and challenges remain significant, Insmed is strategically positioned to explore these complex questions at scale. Ultimately, the viability of synthetic rescue as a pathway forward will depend not only on the data produced but also on the company’s ability to translate these insights into effective treatments.