Our science digs deep, beyond what others overlook, to unearth potential solutions for difficult-to-treat conditions.
What unites our team and inspires our research is a desire to make a profound impact on patients’ lives. We are targeting a wide array of serious and rare diseases for which limited, if any, treatment options exist today. Leveraging cutting-edge technologies and employing new modes of drug discovery, delivery, and development, we seek to bring forward potentially life-transforming therapies that address patients’ most urgent unmet needs.
Our Research Team
Our research team, based in four laboratories across the U.S. and UK, drives innovation across our pipeline—from discovering potential new therapies for hard-to-treat diseases to optimizing formulation and delivery design. Led by world-class experts with a proven track record of breakthrough innovation, the team has extensive experience in biochemistry/biophysics, biology, drug target identification, protein engineering, computational biology, immunology, microbiology, chemistry, aerosol science, bioanalytics, gene therapy, gene editing, artificial intelligence, toxicology, and formulation design. Our researchers leverage a combination of platform technologies and modalities with the hope of offering a distinct advantage in support of our pursuit to deliver much-needed medicines to patients with rare and serious diseases.
We support medical and scientific research conducted by external scientists, clinicians, and organizations that aligns with our areas of interest.
With world-class experts in gene therapy product development at its helm, our San Diego-based research team is working to address many of the existing challenges associated with gene therapy. While gene therapy holds significant promise for transforming serious diseases that previously had no treatments—and is now a validated approach with several approved products—a number of challenges remain, including high dose requirements, increased toxicity with repeat dosing, immunogenicity, and reduced efficacy. The San Diego team’s differentiated approach has the potential to deliver safer and more effective new gene therapies for monogenic diseases in less time and for lower cost. In addition, leveraging the protein deimmunization work taking place at our New Hampshire lab, our researchers in San Diego are exploring how repeat dosing may be feasible for gene therapy without increased toxicity.
Insmed is also building out a state-of-the-art facility in San Diego to support our gene therapy operations and manufacturing. Utilizing breakthrough technology and capabilities, our manufacturing approach has the potential to reduce manufacturing time and cost.
Meet the San Diego Leadership
Chief Scientific Officer
Brian joined the Insmed team in August 2021 when Insmed acquired a gene therapy company that he had co-founded focused on life-threatening and debilitating disorders of the central nervous, musculoskeletal, ocular, and other systems. Brian brings more than 20 years of experience in gene therapy development and manufacturing expertise including the development and commercialization of a gene therapy for Spinal Muscular Atrophy. Brian’s scientific training was at University of San Diego and the prestigious Salk Institute for Biological Studies in La Jolla, CA. Brian served as faculty at The Ohio State University and at Nationwide Children’s Hospital in Columbus, Ohio, as Professor and Endowed Chair of Pediatrics. He has co-authored over 100 scientific publications in high-impact journals with specific expertise in neuroscience and adeno-associated virus gene therapy. Brian is co-inventor on numerous patent applications, which has led to the creation of biotechnology companies including AveXis where he served as Chief Scientific Officer.
With deep expertise in the areas of computational biology, artificial intelligence, biochemistry, protein engineering, and immunology, our New Hampshire-based research team drives the protein deimmunization arm of Insmed’s early-stage research engine.
Utilizing our proprietary Deimmunized by Design® (DbD) platform, the New Hampshire team is focused on re-engineering a wide range of proteins, such as therapeutic proteins, monoclonal antibodies (mAbs), and adeno-associated virus (AAV) capsids, to reduce their innate immunogenicity while maintaining—and potentially enhancing—their function. Applications of this work may include the augmentation of marketed therapies in high unmet need indications where immunogenicity and toxicities limit treatment options, as well as the development of novel gene therapies that may allow for repeat dosing with enhanced efficacy. The platform has the potential to address current challenges in gene therapy.
Leveraging the DbD platform, the New Hampshire team is working to create a library of biotherapeutic agents for internal development, and potentially partnering opportunities, while supporting the gene therapy and gene editing work happening in our San Diego Research labs.
Meet the New Hampshire Leadership
Vice President, Biologics Research
Karl joined Insmed in March 2021 as part of Insmed’s acquisition of the Deimmunized by Design® platform technology, which he and colleague Chris Bailey-Kellogg pioneered at their startup company. Karl has more than 20 years’ experience in biotechnology and biotherapeutics, including more than 15 years as a professor of bioengineering at Dartmouth College and a decade of experience as an entrepreneur, during which he served as CEO of several affiliated startup companies. He has co-authored more than 60 peer-reviewed scientific publications, has received a Coulter Translational Research Award, is a co-inventor on eight published patent families, and in 2021 was inducted into the National Academy of Inventors as a Senior Member. Karl received a bachelor’s degree in chemistry from Texas State University and a doctorate from the University of Texas at Austin, where he studied directed evolution technologies for biomolecular engineering.
Executive Director, Computational Biology
Chris joined Insmed in March 2021 as part of Insmed’s acquisition of the Deimmunized by Design® platform technology, which he and colleague Karl Griswold pioneered at their startup company. At Insmed, he leads the development and application of computationally-driven biomolecular engineering methods in the pursuit of next-generation biotherapeutics. In his former academic career, he attained the rank of Full Professor at Dartmouth, receiving an NSF Career award and an Alfred P. Sloan Foundation fellowship and publishing over 100 peer-reviewed scientific articles. Chris received his bachelor’s and master’s degrees in computer science from MIT and his Ph.D. in computer and information science from Ohio State, and completed postdoctoral training in computational structural biology at Dartmouth.
With experience spanning both biologics and small molecules, our New Jersey-based research team supports Insmed’s early-stage research as well as our mid-stage development. The New Jersey team proudly created our first marketed therapy and one of our mid-stage pipeline candidates in-house and continues to uphold its legacy of leadership in inhalation delivery and formulation, even as we expand into highly innovative new areas of research.
With deep experience in AAV-based gene therapy, protein characterization, and protein production, the team supports the gene therapy work primarily based in San Diego and the protein deimmunization work being conducted in New Hampshire. Early-stage work in gene editing also takes place in New Jersey.
Leveraging its extensive skill set in small molecule research, the team is working to design next-generation DPP1 inhibitor candidates and other small molecule candidates that can complement our work in DPP1 inhibition by reducing neutrophil-mediated inflammation. These efforts use cheminformatics, artificial intelligence, machine learning, and molecular modeling to identify optimal candidates.
The team also conducts critical mechanism-of-action work to support the development of TPIP, which is currently in mid-stage development for forms of pulmonary hypertension.
Lastly, the New Jersey team provides extensive support for Insmed’s business development efforts, evaluating potential opportunities to partner with or in-license external innovations that align with our mission to transform patients’ lives.
Meet the New Jersey Leadership
Chief Technology Officer
Walter joined the Insmed team in December 2010 in connection with the business combination between Insmed and Transave, where he had previously served as Chief Technology Officer. Walter has over 25 years’ experience utilizing nanoparticle drug delivery systems for multiple routes of administration including inhalation. Walter started his pharmaceutical career at The Liposome Company followed by Elan Drug Delivery; he has co-authored 140 scientific publications and presentations and has been co-inventor on over 40 U.S. patent applications. Walter received his bachelor and doctoral degrees in Biophysical Chemistry from the University of Virginia, where he studied liposomes as model membrane systems.
Led by world-class experts in the field of synthetic rescue, Insmed’s Cambridge, UK-based research team focuses on using whole genome assays and human genetics to identify and pursue drug targets for genetic diseases.
Informed by decades of research from the laboratory of synthetic lethality pioneer Professor Sir Steve Jackson, the Cambridge team applies our synthetic rescue platform in an effort to correct the effects of disease mutations and ‘rescue’ cells from disease. By mining the entire human genome for genes that modify disease phenotypes, we are able to provide robust validation of potential new targets that may unlock new ways to treat a broad range of serious and rare diseases.
The synthetic rescue principles that our team is progressing are applicable across a wide range of therapeutic modalities, including small molecules, oligonucleotides, and gene therapies.
Meet the Cambridge Leadership
Chief Research Officer
Sir Steve joined the Insmed team in June 2023 following Insmed’s acquisition of Adrestia Therapeutics, which he co-founded. In addition to his role at Insmed, he is the University of Cambridge Professor of Biology and Senior Group Leader at the Cancer Research UK Cambridge Institute. Before arriving in Cambridge in 1991, he carried out four years’ post-doctoral training in the laboratory of Professor Robert Tjian at UC Berkeley. Sir Steve’s academic laboratory has played pioneering roles in shaping our understanding of DNA repair processes and how defects in them give rise to disease. In addition to conceiving Adrestia, Sir Steve founded and served as Chief Scientific Officer for Mission Therapeutics and KuDOS Pharmaceuticals, the latter of which developed a blockbuster anti-cancer drug.
Sir Steve is a fellow of the Royal Society and the Academy of Medical Sciences UK. He has received various awards, including the 2015 Gagna and van Heck Prize for Medicine, the 2016 King Faisal International Prize for Science, the 2016 AH. Heineken Prize for Medicine, the 2019 Fondation ARC Léopold Griffuel Award for Translational and Clinical Research, and the 2020 Royal Society Mullard Award. In the King’s 2023 Birthday Honours List, he was awarded a knighthood for his services to innovation and research. Sir Steve received his BSc in Biochemistry from the University of Leeds and his PhD in Molecular Biology from the University of Edinburgh.
Senior Vice President and Site Lead
Rob joined the Insmed team in June 2023 following Insmed’s acquisition of Adrestia Therapeutics, where he served most recently as CEO. Prior to Adrestia, Rob was Founder and Chief Business Officer at Affinia Therapeutics, a Boston-based AAV gene therapy company backed by NEA, Atlas, and F-Prime that that focuses on pioneering a shift to a new class of rationally designed gene therapies to treat rare and prevalent diseases.
Prior to Affinia Therapeutics, he was Founder, President, and Managing Partner of Alacrita, a transatlantic management consulting firm specializing in corporate strategy and business development for the biopharma industry. Over 10 years at Alacrita, Rob led or oversaw consulting projects across several therapeutic areas, serving biopharma clients in the U.S., EU, and beyond. Before this, he held multiple roles in business development at Onyvax, an SR One and 3i portfolio company developing cell-based products to treat cancer. Rob received his BS Hons in Molecular Cell Biology from the University of Nottingham.
Senior Vice President, Research
Tom joined the Insmed team in June 2023 following Insmed’s acquisition of Adrestia Therapeutics, where he served most recently as Chief R&D Officer. He is a leading medicinal chemist with broad experience in drug discovery. Over the course of his career, he has implemented transformative innovation in drug discovery and delivered multiple programs into the clinic. Previous roles include VP Global Head of Oncology Chemistry at AstraZeneca, VP & Head of Medicinal Chemistry at Astex Pharmaceuticals, Principal Investigator at the Structural Genomics Consortium in Oxford University, and Head of Lead Discovery for the Neurology Centre of Excellence at GlaxoSmithKline. He has made scientific and leadership contributions to the discovery of over a dozen pre-clinical drug candidates, of which seven have progressed to IND and five to Phase 2 trials thus far.
Tom is a Fellow of the Royal Society of Chemistry, having served on the Biological and Medicinal Chemistry Sector committee and the Chemistry-Biology Interface Division Council, and has authored more than 100 publications and patents. Tom obtained his Chemistry MA at Oxford University and his PhD at the ETH Zürich with Prof A. Vasella.