Addressing significant unmet patient needs
At Insmed, we are advancing clinical development programs that have the potential to make a significant impact on their respective disease landscapes. Every program we bring forward is driven by our desire to address significant patient needs that are not adequately being met today.
Amikacin Liposome Inhalation Suspension
We’re investigating amikacin liposome inhalation suspension for the treatment of NTM lung disease caused by MAC in a frontline setting of newly diagnosed patients.
We’re investigating brensocatib, a small molecule, oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP1), as a potential treatment for neutrophil-mediated diseases, including bronchiectasis and cystic fibrosis. Brensocatib is an investigational product that has not been approved for any indication in any jurisdiction.
We’re investigating treprostinil palmitil inhalation powder (TPIP), a dry powder formulation of treprostinil palmitil, as a potential therapy for rare pulmonary disorders, including pulmonary hypertension associated with interstitial lung disease and pulmonary arterial hypertension. TPIP is an investigational product that has not been approved for any indication in any jurisdiction.
We’re advancing an early-stage research engine encompassing a wide range of technologies and modalities, including gene therapy, artificial intelligence-driven protein engineering, and protein manufacturing. These efforts will fuel our pipeline for years to come, enabling us to develop novel and cutting-edge technologies that may address a broad range of rare diseases across therapeutic areas.
Our Research Team
Our research team, based in three U.S. Research and Development Labs (RDLs), 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, 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.
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 RDL-SD 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 RDL-NH, 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 RDL-SD 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 RDL-NH 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 RDL-NH 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.
Learn about the research being led by RDL-NH.
Meet the RDL-NH Leadership
Executive Director, 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 RDL-NJ 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 RDL-NJ 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 RDL-NJ 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 RDL-NJ 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 RDL-NJ 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.