Our Mission
The Institute for Translational Medicine and Therapeutics (ITMAT) supports research at the interface of basic and clinical research focusing on developing new and safer medicines. ITMAT supports faculty, includes dry and wet research space and amongst its centers and programs are the Penn Medicine Biobank, the Investigational Drug Service and the Center for Human Phenomic Science (CHPS). CHPS is present in both Penn and the Children's Hospital of Philadelphia (CHOP). ITMAT issues a series of funding calls to support interdisciplinary, translational research including that which crosses the pediatric to adult divide. ITMAT also offers an extensive palette of educational programs in clinical and translational science both for investigators and research support staff.
Our Objectives
The objectives of ITMAT are:
- To cluster, expand, and democratize access to the resources relevant to the conduct of translational medicine and therapeutics and to sustain and expand a visible home for the emerging discipline of human phenomic science;
- To increase the number of investigators pursuing translational research and educate the workforce through novel educational programs and targeted recruitments;
- To identify and minimize the obstacles faced by investigators conducting clinical and translational research;
- To enhance the clinical implementation of the discoveries of translational research.
ITMAT includes investigators from all schools at Penn, the Children's Hospital of Philadelphia, and the Wistar Institute. Amongst its sources of funding, ITMAT is the academic home for the NIH funded Clinical and Translational Science Award.
Our Focus Areas
The focus areas of ITMAT are:
- Translational Therapeutics with an emphasis on integrating preclinical science in cells and model systems with mechanistic elucidation of drug action and variability in drug response in humans and
- Bridging the pediatric to adult divide.
About ITMAT
Started in 2005 as the world's first institute dedicated to translational medicine, ITMAT has grown into a multidisciplinary network of ~2,500 investigators spanning Penn, the Children's Hospital of Philadelphia (CHOP), and other partner organizations.
ITMAT sponsors workshops on a variety of topics pertaining to translational science and has held annual international meetings each October. The 21st such meeting entitled "Intermezzo; 20 Years of Translational Therapeutics at PENN and CHOP" will be held on Monday, October 5, 2026. See full symposium details.
ITMAT meetings have also been held abroad in partnerships with the Universities of Edinburgh and Kyoto.
ITMAT's Impact
Over the past 20 years, the Institute for Translational Medicine and Therapeutics (ITMAT) at the University of Pennsylvania has helped transform Penn into one of the world's leading centers for translational science, bridging the gap between laboratory discovery and patient care. Founded in 2005 as the world's first institute dedicated to translational medicine, ITMAT has grown into a multidisciplinary network of approximately 2,500 investigators spanning Penn, the Children's Hospital of Philadelphia (CHOP), and other partner organizations. Through its support of core research infrastructure, pilot funding, education, and collaboration, ITMAT has created an environment where discoveries can move more rapidly from basic science to clinical application.
ITMAT's impact is reflected in many of Penn Medicine's most significant biomedical advances. By serving as the academic home of Penn's NIH-funded Clinical and Translational Science Award (CTSA) program and supporting resources such as the Penn Medicine Biobank (PMBB), the Center for Human Phenomic Science (CHPS), and the Investigational Drug Service (IDS), and a repertoire of educational offerings in translational science ITMAT has enabled investigators to pursue innovative therapies across a wide range of diseases. The Institute has fostered a culture of collaboration between adult and pediatric medicine and has contributed to breakthroughs in gene therapy, cell therapy, precision medicine, and most recently gene-editing approaches for rare genetic disorders. Over two decades, ITMAT has not only strengthened research capacity at Penn and CHOP but has also helped establish Philadelphia as a global hub for translational therapeutics and the development of new and safer medicines.
While ITMAT itself is an enabling institute rather than a drug-development company, it has provided infrastructure, funding, education, collaborations, clinical research support, and CTSA ecosystem that helped many of Penn and CHOP's breakthrough therapies reach patients. Notable examples include:
- CAR-T cell therapy for leukemia and lymphoma: Penn investigators led by Carl June pioneered the first FDA-approved CAR-T therapy, in which a patient's own immune cells are genetically engineered to attack cancer. This work helped establish an entirely new class of cancer treatment and has benefited thousands of patients worldwide. ITMAT's translational infrastructure helped support the environment in which these discoveries moved from laboratory studies to clinical implementation.
- Luxturna® (voretigene neparvovec): Developed by Penn and CHOP scientists Jean Bennett, Albert Maguire, and Katherine High, Luxturna became the first FDA-approved gene therapy for an inherited disease. It restores functional vision in many patients with blindness caused by RPE65 mutations and created a regulatory and scientific roadmap for the modern gene therapy field.
- In vivo gene-editing therapies: More recently, Penn and CHOP investigators, Kiran Musunuru and Rebecca Ahrens–Nicklas developed personalized CRISPR-based treatments for ultrarare disorders, including a landmark custom gene-editing therapy for an infant with CPS1 deficiency, demonstrating the feasibility of rapidly creating individualized genetic medicines.