Susan Furth, Executive Vice President and Chief Scientific Officer, Children’s Hospital of Philadelphia

In an interview with Invest:, Susan Furth, executive vice president and chief scientific officer of Children’s Hospital of Philadelphia, discussed the future of pediatric research, including partnerships, equity in clinical trials, and workforce development. “That mission-driven focus is what allows us to persevere, even in uncertain times,” said Furth.

What major shifts have you seen in pediatric biomedical research over the past year, and how have they affected your priorities?
The past year has been an unusual departure from business as usual. Administrative changes and evolving approaches to research funding at the National Institutes of Health (NIH) introduced significant uncertainty, particularly for organizations like ours focused on children’s health in Philadelphia and beyond.

NIH funding supports most research into childhood conditions and adult diseases with pediatric origins, such as heart disease, kidney disease, and cancer. At Children’s Hospital of Philadelphia, about 40% of our external research funding comes from the NIH. That support has driven many discoveries and life-changing treatments.

This year brought disruptions. Some grants were canceled, though many were later reinstated. While NIH activity temporarily slowed, we’ve recently seen an encouraging increase in new awards. Our investigators showed remarkable adaptability, securing foundation and philanthropic funding to maintain momentum.

One of CHOP’s great strengths is our people. We have scientists and clinicians who are passionately committed to using science to solve real-world challenges for children and families. That mission-driven focus is what allows us to persevere, even in uncertain times.

What are the most promising developments in cell and gene therapy for children, and how is CHOP helping to advance those treatments?
CHOP and the Philadelphia region have long been at the forefront of cell and gene therapy, including early breakthroughs in CAR-T cell therapy and most recently, personalized gene editing. Our researchers have helped develop cell and gene therapy treatments for cancer and certain forms of blindness, often in partnership with colleagues at the University of Pennsylvania.

We’ve also advanced therapies for sickle cell disease and contributed to vaccine development. Beyond science, these innovations have helped fuel economic growth in Philadelphia and across Pennsylvania.

One compelling example in the cell and gene therapy space is baby KJ. He was diagnosed with a rare genetic disorder thanks to a newly developed rapid genetic test in the first few days of life, and the insight of an astute CHOP clinician. Decades of research at CHOP and Penn, and recent work led by Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunuru, enabled the creation of a personalized gene-editing therapy for KJ.

Without this therapy, KJ’s only option would have been a liver transplant, which he was too small to undergo. Instead, he received a treatment tailored to his condition, and early signs suggest it is working.

KJ’s story shows how sustained scientific investment can lead to transformative care. While his was the first case, the broader impact is just beginning. This kind of work holds enormous potential to treat or even cure many more diseases.

How are emerging technologies changing how discoveries are made and translated into care for children?
Technology influences nearly every aspect of our research. In KJ’s case, the breakthrough diagnosis was possible because of our ability to process and interpret large-scale data.

We’ve made significant investments in genomic sequencing and built systems to manage and analyze this data. With patient and family consent, we collect biological samples and search for disease-causing patterns. Using tools like artificial intelligence, our teams can extract insights into how diseases operate at the molecular level.

Looking ahead, we see an opportunity to connect clinical, genomic, and biological data to develop a full picture of a child’s disease — again, only with consent. This kind of integration supports the development of personalized therapies, much like the one that helped KJ.

While many of these technologies are still advancing, the future is clear: Pediatric care will increasingly depend on the ability to understand and respond to each child’s unique biological profile.

What approaches are helping improve equity in clinical research and trial design?
At CHOP, advancing research-driven precision medicine means ensuring it is accessible to everyone. Equity is a key priority in how we design and implement clinical trials.

To support this, we launched the Health Equity Research Methods Initiative. This team helps ensure trials are inclusive, from how materials are written to how patients are recruited and supported, regardless of where they live or their access to transportation.

We’re also expanding opportunities for care and research participation from home. Studies increasingly allow patients to engage remotely through video interviews, text messages, or at-home lab work in partnership with outside organizations.

It’s also essential that research participants reflect the community of children we serve. For observational studies, we strive to include families from a wide range of backgrounds and circumstances.

One silver lining of the pandemic was discovering how much can be done remotely. That flexibility has helped us reach more families and include them in meaningful research.

Ultimately, our goal is to ensure that both the research we conduct and the care we deliver are accessible to every child and family who could benefit.

How do partnerships with Penn and industry help CHOP move discoveries from the lab to patient care more quickly?
A key priority for CHOP Research Institute is strengthening partnerships, because bringing a lab discovery to patient treatment is a long and complex process. One notable recent example of a strong partnership is the Lurie Autism Institute.

Philadelphia Eagles Chairman and CEO Jeffrey Lurie, his family, the Nancy Lurie Marks Family Foundation, and the Lurie Family Foundation contributed $50 million to Penn and CHOP to establish the Institute. This transformational gift will enable researchers to better understand autism’s biological basis, accelerate early diagnosis, and advance treatment. CHOP focuses on children and young adults and on translational research, while Penn focuses on older populations and has great strengths in basic science, creating a powerful and complementary lifespan research partnership.

Another example of a strong partnership is the CHOP-led Patient-Centered Outcomes Research Institute (PCORI)-funded consortium, PEDSnet, a consortium of children’s hospitals. This collaboration brings together up to 12 children’s hospitals to share data and insights on both rare and common conditions. By pooling cases, researchers can evaluate treatments and long-term outcomes, and by partnering with patients and families, develop research agendas that truly reflect the concerns of affected families.

How is CHOP preparing the future workforce of investigators, and what skills are most important?
Training the next generation of scientists and physicians is one of CHOP’s core missions and a major strategic initiative, even amid funding uncertainties.

We are committed to creating equitable access to research careers, with training opportunities that reflect the diversity of the children we serve. Through CHOP Research Institute’s Office of Academic Training and Outreach Programs, we offer programs for students from elementary school through college. Our summer science program, for example, gives younger students hands-on lab experience and a window into future careers.

Beyond fostering curiosity, we emphasize collaboration. Science is a team effort. Trainees must be comfortable working across disciplines, whether it’s combining biology with computational tools or linking clinical insight to drug development. This kind of interdisciplinary collaboration is essential to solving pediatric health challenges.

What are CHOP’s top priorities for the next three to five years?
Our overarching goal of improving the lives of children both locally and globally remains the same. To achieve this goal, we are focused on several priorities. One is training the next generation of physician-scientists and investigators. Supporting early-career professionals is essential for maintaining curiosity and commitment, especially during times of uncertainty.

Another priority is continuing our progress in personalized gene editing and cell and gene therapy. Building on breakthroughs like the therapy developed for baby KJ, we are working with partners at Penn and other academic centers to pioneer new therapeutic technologies that can address previously untreatable diseases.

We are also investing in data and artificial intelligence tools to accelerate discovery. By improving our ability to analyze and interpret large-scale data, we aim to move research forward more efficiently and deliver meaningful benefits to children as quickly as possible.

At the same time, we recognize that CHOP plays a vital role in the broader Philadelphia community. We are a major regional employer, with more than 29,000 workforce members. In 2025, CHOP was recognized by Forbes as one of America’s best large employers. Beyond healthcare, CHOP has an annual economic impact of more than $9.4 billion in our region. This makes the region a strong foundation for continued investment, innovation, and growth.

How is CHOP expanding its research infrastructure to support future scientific innovation?
CHOP has an expanding research presence that now spans the Schuylkill River and includes four research buildings. The newest, the Morgan Center for Research and Innovation, is a 17‑story, 350,000-square‑foot facility that houses both wet labs for bench research and dry labs for computational and data-driven work. It is designed to enhance synergies between experimental and computational research.

The building also features integrated collaborative spaces, including wet and dry lab adjacency, lecture areas, and an innovation “Idea Lab” on the first floor to support development of new tools and prototypes, such as custom-made microscopes, and novel devices, such as breath-based infectious disease diagnostics.