CHOP Research Poster Day and Scientific Symposium: Celebrating Frontier Programs

2025 CHOP Poster Day and Scientific Symposium

Scientists, staff, and students from CHOP and the University of Pennsylvania community celebrated a packed Poster Day and spirited Scientific Symposium event May 6, 7, and 8. While the traditional poster sessions gave trainees a chance to share their research, a four-part lecture series celebrated the remarkable breakthroughs achieved over the last decade by CHOP's Frontier Programs. The series highlighted CHOP's 27 Frontier Programs through the lens of four themes: novel therapeutics, innovating diagnosis, engineering medical devices, and leveraging omics for precision medicine.

"This event, now in its 32nd year, always brings me joy," said Susan Furth, MD, PhD, Executive Vice President and Chief Scientific Officer at CHOP Research Institute. "I get to catch up with faculty, staff, and students, from CHOP and the University of Pennsylvania, while learning about exciting new pediatric research."

What began as a bold pilot initiative in 2015 has grown into a globally recognized portfolio of 27 Frontier Programs — each redefining what's possible in pediatric medicine. These programs now care for nearly 32,000 children annually, offering therapies, diagnostics, and research opportunities that are often the first or only of their kind in the nation.

Access the video transcript

While the leaders of each Frontier Program are listed below, the symposium featured a wide range of voices from across the programs. During each of the four sessions, presenters shared how their initiatives are driving advances in pediatric research and care at CHOP and around the world.

From Bench to Bedside: Accelerating Novel Therapeutics for Pediatric Care

Frontier Program leaders participated in a PDSS session focused on novel therapeutics.

Minds Matter Concussion Program
(Christina Master, MD; Kristy Arbogast, PhD)

This Frontier Program has become the leading concussion care center in the region through exceptional clinical assessment and advanced research in the field. The International Consensus Statement on Concussion in Sport incorporates Minds Matter's visio-vestibular examination as a key tool in evaluating pediatric concussions. Read our Cornerstone story to learn more about the Minds Matter Concussion Program.

Gene Therapy for Inherited Metabolic Disorders
(Rebecca C. Ahrens-Nicklas, MD, PhD; Can Ficicioglu, MD, PhD)

The program is pioneering novel gene therapies for inherited metabolic disorders and establishing a world-class clinical program dedicated to the delivery of novel therapies to patients . The program is focused on developing liver-directed gene-editing therapies for phenylketonuria and multiple sulfatase deficiency. Read about related research in Cornerstone.

Sickle Cell and Red Cell Disorders Curative Therapy Center (CuRED)
(Stefano Rivella, PhD; Janet Kwiatkowski, MD; Timothy Olson, MD)

The CuRED team aims to change care pathways for red blood cell disorders through the development and delivery of novel gene therapies and stem cell transplants to patients. They demonstrated that gene editing tools can be delivered via lipid nanoparticles, which could enable researchers to modify diseased blood cells in patients with fewer side effects. Learn more about the CuRED Frontier Program in Cornerstone.

Novel Therapeutics for Bleeding Disorders (NoT Bleeding)
(Lindsey A. George, MD; Rodney Camire, PhD; Benjamin J. Samelson-Jones, MD, PhD; Leslie Raffini, MD, MSCE; Bhavya Doshi, MD)

The Novel Therapeutics for Bleeding Disorders (NoT Bleeding) Frontier Program develops treatments for rare bleeding disorders, establishing a national referral center and exploring repurposed drugs, antibodies, and gene therapies. The program conducted research that aided in the development emicizumab, a medication used to treat hemophilia A, or similar bi-specific antibodies for hemophilia B therapy. Learn more about the NoT Bleeding program in Cornerstone.

Congenital Diaphragmatic Hernia Program
(Holly Hedrick, MD; Emily Partridge, MD, PhD)

As one of the largest Congenital Diaphragmatic Hernia (CDH) treatment centers in the country, this program aims to become the international epicenter for patient care and research innovation for CDH. CHOP is an FDA site of record for the FETO trial, a safety trial for the use of prenatal tracheal occlusion for the most severe CDH patients.

Susan S. and Stephen P. Kelly Center for Cancer Immunotherapy
(Stephan Grupp, MD, PhD)

This Frontier Program was established with the goal of making chimeric antigen receptor (CAR) T-cell therapy an accessible form of cancer treatment for childhood acute lymphoblastic leukemia (ALL). In the years since this frontier began, six CAR T-cell therapies have been approved by the FDA, starting with ALL and expanding to other cancer types. Read our Cornerstone story to learn more about the Cancer Immunotherapy program.

Rare Lung Diseases Center
(Lisa R. Young, MD; Sharon McGrath-Morrow, MD, MBA)

The Center for Rare Lung Diseases Frontier Program aims to improve diagnostics by expanding genetic and pathologic testing. It will identify genetic targets, develop biomarkers, and model molecular mechanisms to enhance patient outcomes. Learn more about the Rare Lung Diseases Center in Cornerstone.

Innovating Diagnosis: Advancing Translational Pathways for Cutting-Edge Diagnostics

Frontier Program leaders participated in a session highlighting cutting-edge diagnostics.

Center for Precision Medicine for High-Risk Pediatric Cancer
(Theodore Laetsch, MD; Marilyn M. Li, MD; Adam C. Resnick, PhD; Kai Tan, PhD)

The center builds upon ongoing precision medicine efforts across CHOP departments to establish a "one-stop" clinic for high-risk patients, develop state-of-the-art molecular tumor profiling, and build the largest multi-omics database for pediatric cancer. A study published in December 2024 found that the precision medicine larotrectinib was highly effective as a frontline therapy in certain pediatric patients with newly diagnosed solid tumors, sparing the need for traditional chemotherapy. Read more in this Cornerstone story.

Food Allergy Center
(Jonathan M. Spergel, MD, PhD; Terri Brown-Whitehorn, MD; David A. Hill, MD, PhD)

The Food Allergy Center is the only program with national expertise in all four types of food allergy. The program is developing novel diagnostic testing, specifically investigating the role of food specific T-cells in Eosinophilic Esophagitis (EoE), to guide early dietary interventions and potential food re-introductions in patients with EoE by detecting elevated milk-dependent memory T-cells and other immune markers.

Thyroid Center
(Andrew J. Bauer, MD; Sogol Mostoufi-Moab, MD, MSCE; Aime T. Franco, PhD)

The Thyroid Center is renowned for its treatment program, while also being the only center in the country with a supporting translational research program. This Frontier Program successfully launched the Child & Adolescent Thyroid Consortium, which has led to research innovations in multiple national clinical trials studying pediatric thyroid cancer. Read our Cornerstone story to learn more about the Thyroid Center.

CHOP Kidney Stone Center
(Gregory E. Tasian, MD, MSc, MSCE; Michelle Denburg, MD, MSCE; Stephen R. Master, MD, PhD)

The Pediatric Kidney Stone Center was created to transform how kidney stone disease is diagnosed, treated and prevented. The program is leveraging multi-omics research, data science, and machine learning to develop a more accessible and comprehensive urine test that can provide earlier detection and detailed insights into kidney stone disease. Read our Cornerstone story to learn more about the CHOP Kidney Stone Center.

Center for Precision Diagnosis and Treatment of Motility Disorders
(Hayat Mousa, MD; Robert Heuckeroth, MD, PhD; Alain J. Benitez, MD, MSTR)

The Center for Precision Medicine for Diagnosis and Therapy for Pediatric Motility Disorders Frontier Program targets clinical and research needs for children with chronic intestinal pseudo-obstruction, Hirschsprung's disease, gastroparesis, and achalasia. A focus has been translating new technologies for treatment and diagnosis, including EndoFLIP/EsoFLIP for radiation-free esophageal dilation and surgical therapy outcome assessment.

Advancement of Hyperinsulinism Care and Research
(Diva DeLeon-Crutchlow, MD)

This program uses personalized medicine to treat hyperinsulinism (HI), a neonatal condition in which insulin dysregulation results in dangerously low blood sugar levels and potential brain damage. Researchers built a patient data collection platform to develop more accurate diagnostic tools and aim to accelerate late-stage clinical trials for an HI-ameliorating compound, including the publication of global HI guidelines. Learn more about the Advancement of Hyperinsulinism Care and Research Frontier Program in Cornerstone.

Engineering the Future: Transforming Patient Care Through Novel Medical Devices

Frontier Program leaders joined a session featuring transformative medical devices.

Delivery Room of the Future
(Elizabeth Foglia, MD, MA MSCE; Anne M. Ades, MD; Natalie Rintoul, MD; Thomas Reynolds)

The Delivery Room of the Future Program aims to inform resuscitation guidelines specific for infants with congenital anomalies through data analysis and innovation. An observational study on respiratory function after birth in infants with congenital diaphragmatic hernia was the first to characterize respiratory mechanics during lung aeration after birth among infants with this condition. Learn more about the Delivery Room of the Future in Cornerstone.

Mitochondrial Medicine Program
(Marni J. Falk, MD; Amy Goldstein, MD)

The Mitochondrial program seeks to develop diagnostic genetic, biochemical, and tissue testing, and precision therapies to treat mitochondrial disease. Leading a study with the University of Pennsylvania, they plan to examine racial disparities when diagnosing mitochondrial diseases. Read our Cornerstone story to learn more about the work of the Mitochondrial Medicine Frontier Program.

Wyss/Campbell Center for Thoracic Insufficiency Syndrome
(Patrick J. Cahill, MD; Jason B. Anari, MD)

Building on the development of a pioneering device to help expand the chest, this program focuses on developing novel imaging and new devices to care for children with thoracic insufficiency syndrome. The Frontier Program conducted a clinical trial exclusively at CHOP that led to FDA approval of a novel device for treating patients with adolescent idiopathic scoliosis. Read our Cornerstone story to learn more about the Center for Thoracic Insufficiency Syndrome.

Biomedical Optical Devices to Monitor Cerebral Health
(Todd Kilbaugh, MD; Wesley B. Baker, PhD)

This Frontier Program seeks to develop a first-of-its-kind noninvasive device that monitors brain oxygen levels. In a preclinical study, findings showed that measurements taken with this innovative neurometabolic optical monitoring technology, during extracorporeal membrane oxygenation , can predict brain outcome. Read more about the Biomedical Optical Devices to Monitor Cerebral Health Frontier Program in Cornerstone.

Center for Pediatric Airway Disorders
(Ian Jacobs, MD; Karen Zur, MD)

The Pediatric Airways Disorders Program is one of the few centers in the country specializing in tracheal reconstruction, recurrent laryngeal nerve reinnervation, and a variety of other specialized airway procedures. A new research innovation involves a novel biomaterial, known as MEND, to innovate airway reconstructions and other implant surgeries. Read our Cornerstone story to learn more about the Center for Pediatric Airway Disorders.

Jill and Mark Fishman Center for Lymphatic Disorders
(Yoav Dori, MD, PhD)

This program has pioneered new imaging tools and a minimally invasive catheterization technique to identify and treat lymphatic leaks and lymphatic flow disorders. Research innovations include imaging advancements, updates to surgical management, ocular lymphatics, and lymphatic involvement in kidney health, gastrointestinal diseases, and the brain. Read our Cornerstone story to learn more about the Center for Lymphatic Imaging and Intervention

Topolewski Center for Pediatric Heart Valve Disorders
(Jonathan Chen, MD; Michael Quartermain, MD; Matthew Gillespie, MD; Matthew A. Jolley, MD; Robert J. Levy, MD)

This program aims to coordinate care for pediatric valve patients, offer personalized valve therapies, and define therapeutic drug targets to mitigate the progression of the disease. Dr. Gillespie led three multi-site clinical trials that analyzed the Harmony Transcatheter Pulmonary Valve™ (TPV), a device used for the treatment of severe pulmonary regurgitation in right ventricular outflow tract, resulting in the device's FDA approval. Read this Cornerstone story to learn more about the Frontier Program.

Decoding Disease: Leveraging Omics and Big Data for Precision Medicine

Frontier Program leaders participated in a session focused on the power of omics and big data for precision medicine.

Comprehensive Vascular Anomalies Program
(Denise Adams, MD; Hakon Hakonarson, MD, PhD)

This program comprises a team of multidisciplinary clinicians and researchers working to discover the underlying genetic mutations that cause debilitating complex vascular anomalies and identify targeted curative therapies. They developed liquid biopsy-based diagnostic techniques to identify previously undescribed genotype-phenotype associations and guide medical therapy in individuals with vascular anomalies. Read about the Comprehensive Vascular Anomalies in Cornerstone.

Center for Pediatric Inflammatory Bowel Disease
(Robert Baldassano, MD; Andrew Grossman, MD; Judith Kelsen, MD; David Piccoli, MD)

This Frontier Program focuses on novel research to develop more targeted, effective treatments for the more than 2,400 patients affected by inflammatory bowel disease who visit CHOP every year. The Frontier Program is conducting multi-omics studies to identify causative defects and therapeutic targets for precision medicine approaches to care. A VEO-IBD genetic panel was developed and launched, defining 98 known causative genes for VEO-IBD, and available through the Division of Genomic Diagnostics. Learn more about working with organoid models in this Cornerstone story.

Newborn and Infant Chronic Lung Disease Program
(William W. Fox, MD)

The Lung Disease program tests a novel therapy that involves filling the lungs with a special anti-inflammatory liquid with unique gas exchange properties. They filed a provisional patent application for a noninvasive device to deliver this liquid to newborns with respiratory distress syndrome.

Epilepsy NeuroGenetics Initiative (ENGIN)
(Ethan Goldberg, MD, PhD; Ingo Helbig, MD; Mark Fitzgerald, MD, PhD)

ENGIN aims to expand genetic testing to all children with epilepsy to optimize their care and fuel precision therapy development. The ENGIN team collaborated with an industry partner to launch a first-of-its-kind Phase 1b clinical trial for patients with a rare genetic form of epilepsy called myoclonus epilepsy and ataxia due to potassium channel mutation, for which there are no effective treatments. Learn more about ENGIN's research in this Cornerstone story.

Immune Dysregulation Program
(Edward M. Behrens, MD; Kathleen E. Sullivan, MD, PhD; Michele Lambert, MD, MSTR; David Teachey, MD)

This Frontier Program's multidisciplinary team investigates the genetic causes underlying immune dysfunction and accelerates the development of new therapies. The program launched a quality improvement project to ensure appropriate antimicrobial prophylaxis for immunocompromised patients, regardless of income or language preference. Read our Cornerstone story to learn more about the Immune Dysregulation program.

Craniofacial Program
(Eric C. Liao, MD, PhD; Jesse Taylor, MD; William Parenteau, MD)

This Frontier utilizes genomic medicine to develop precision treatment plans and predict outcomes for children with craniofacial conditions. Innovation in craniofacial care will aid in developing novel treatment options, with projects that include multimodal and functional genetic analysis and new molecular therapies. Read our Cornerstone story to learn more about the Craniofacial Center.

Advanced Cardiac Therapies for Heart Failure Patients
(Joseph Rossano, MD, MS; Katsuhide Maeda, MD, PhD; Zoltan Arany, MD, PhD)

The Heart Failure Frontier Program is a leader in pediatric heart failure discovery, prevention, and treatment. It is pioneering an Intensive Rehabilitation and Developmental Intensive Program, improving clinical and neurodevelopmental outcomes for patients on VADs, and with heart transplants. They are studying heart failure progression and the restoration of ventricular function once heart failure develops, through multi-omics studies. Read more in this Cornerstone story.

Featured Speakers

Two distinguished leaders in biomedical research gave featured lectures. William A. Gahl, MD, PhD, Senior Investigator at the National Human Genome Research Institute and Director of the NIH Undiagnosed Diseases Program, delivered an inspiring talk that resonated with CHOP's shared commitment to uncovering the genetic roots of rare and elusive diseases. Dr. Gahl's lecture reminded the audience of the importance of perseverance and hope in tackling the most complex medical conditions.

Mark C. Fishman, MD, Professor of Stem Cell and Regenerative Biology at Harvard and founder of the Pathways Clinical Service at Massachusetts General Hospital, shared his groundbreaking work on chronic diseases, illustrating how discoveries in the lab can illuminate new therapeutic targets and deliver hope to patients for whom few or no treatments previously existed.

This event was made possible through the thoughtful leadership of the Planning Committee —Sharon McGrath-Morrow, MBA, MD and Eric D. Marsh, MD, PhD (Co-Chairs), Edward M. Behrens, MD, Christina L. Master, MD, FAAP, CAQSM, FACSM, FAMSSM, and Benjamin J. Samelson-Jones, MD, PhD — along with partners from Frontier Program Operations and Strategy, and with programmatic support from the Offices of Faculty Development and Academic Training and Outreach Programs. Together, their efforts brought forward key voices to highlight the remarkable breakthroughs CHOP researchers have achieved over the past decade.