August 24, 2026

PARADIGM Personalized Genetic Medicine Symposium

Advancing Precision Gene Editing Therapies

St. Jude Children's Research Hospital, Memphis, TN

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About the meeting

The PARADIGM Personalized Genetic Medicine Symposium was held on August 24, 2026, at St. Jude Children’s Research Hospital. This symposium brought together leading experts in hematology, genome engineering, ethics, and clinical translation to advance the future of individualized therapies for inherited bone marrow failure (BMF) disorders. BMF disorders lead to life-threatening cytopenia, immunodeficiency, and leukemia risk.

Driven by the mission of the St. Jude PARADIGM (Partnership to Advance the Development of Individualized Genomic Medicines) program for rare, life-threatening hematological diseases, the symposium explored emerging scientific discoveries in personalized genomic medicines. Sessions addressed challenges in developing n-of-1/few genomic medicines, and the ethical and regulatory considerations required for responsible clinical implementation.

This full-day event began with joint strategic discussions between St. Jude’s internal advisory committee and the external Scientific Advisory Board. The afternoon will feature an open session with scientific presentations from renowned expert investigators. By fostering cross-disciplinary dialogue, the symposium aimed to align scientific innovation with clinical needs. The ultimate goal is to accelerate safe, effective, and personalized genetic therapies for children with rare blood disorders, reinforcing St. Jude’s mission in transforming the future of treatment of catastrophic pediatric disorders.

Video replay: Afternoon session

Overview

Event: PARADIGM Personalized Genetic Medicine Symposium

Date: August 24, 2026

Location:
St. Jude Children's Research Hospital
Memphis, Tennessee, USA

Department host: Hematology

Hosts:
Shengdar Tsai, PhD
Associate Member, St. Jude Faculty
Department of Hematology

Marcin Wlodarski, MD, PhD
Associate Member, St. Jude Faculty
Department of Hematology

Senthil Bhoopalan, MBBS, PhD
Assistant Member, St. Jude Faculty
Department of Bone Marrow Transplant and Cellular Therapy

Mitch Weiss, MD, PhD
Member, St. Jude Faculty
Chair, Department of Hematology
Arthur Nienhuis Endowed Chair in Hematology

Event contact: paradigm2026@stjude.org

Agenda

1:00-4:00 p.m., Marlo Thomas Center Auditorium
Open to all St. Jude
Time	Presenter/Session
1:05 – 1:30 p.m.	Charlotte Niemeyer — Development of New Therapies for Juvenile Myelomonocytic Leukemia: Old vs. New
1:30 – 1:55 p.m.	Akiko Shimamura — Clonal Surveillance and Outcomes in Shwachman Diamond Syndrome
1:55 – 2:20 p.m.	Timothy Olson — HSCT for Inherited Bone Marrow Failure Syndromes (DBA)
2:20 – 2:35 p.m.	Break
2:35 – 3:00 p.m.	Fyodor Urnov — Personalized Genetic Cures: Innovations from the IG
3:00 – 3:25 p.m.	Winston Yan — Sustainability Models for Interventional Genetics: Why routine drug development and reimbursement will not work
3:25 – 3:50 p.m.	Tami John — Transforming Beta-Thalassemia: The Evolution of Gene TherapiesI
3:50 – 4:00 p.m.	Closing Remarks
4:00 – 5:00 p.m.	Campus Tour for SAB Members

External Scientific Advisory Board Members

Name / Credentials	Current Institution	Current Role / Affiliation
Prof. Charlotte Niemeyer, MD	Medical Center – University of Freiburg, Germany	Professor of Pediatrics; Pediatric Hematology & Oncology.
Akiko Shimamura, MD, PhD	Dana-Farber Cancer Institute / Boston Children’s Hospital; Harvard Medical School	Professor of Pediatrics, Harvard Medical School; Director, Bone Marrow Failure and Myelodysplastic Syndrome Program.
Tami John, MD	Stanford Medicine / Lucile Packard Children’s Hospital Stanford	Clinical Associate Professor, Pediatric Hematology-Oncology
Donald B. Kohn, MD	UCLA	Distinguished Professor of Microbiology, Immunology & Molecular Genetics; Pediatrics/Hematology-Oncology; Molecular & Medical Pharmacology.
Timothy S. Olson, MD, PhD	Children’s Hospital of Philadelphia (CHOP) / University of Pennsylvania	Associate Professor of Pediatrics (Oncology); attending physician in Bone Marrow Failure and Cell Therapy/Transplant.
Fyodor Urnov, PhD	University of California, Berkeley / Innovative Genomics Institute (IGI)	Professor of Molecular Therapeutics; Director for Therapeutic R&D at IGI.
Timothy W. Yu, MD, PhD	Boston Children’s Hospital / Harvard Medical School	Attending Physician, Division of Genetics & Genomics; Associate Professor of Pediatrics, Harvard Medical School.
Julia Vitarello	Mila’s Miracle Foundation	Founder & CEO; co-founder of the N=1 Collaborative and EveryONE Medicines.
Winston Yan, MD, PhD	Broad Institute	Senior Director, Center for Therapeutic Genetics; founding president of the N=1 Collaborative.

Internal Advisory Board Members

Paul Taylor, MD, PhD Member, St. Jude Faculty / Executive Vice President / Scientific Director / Chair, Cell & Molecular Biology Department / Director, Pediatric Translational Neuroscience Initiative / Edward F. Barry Endowed Chair in Cell and Molecular Biology

Ellis Neufeld, MD, PhD Member, St. Jude Faculty / Executive Vice President / Clinical Director / Physician-in-Chief / John and Lorine Thrasher Endowed Chair in Pediatric Medicine

Clifford Takemoto, MD Member, St. Jude Faculty / Director, Clinical Hematology / Lemuel Diggs Endowed Chair in Sickle Cell Disease

Stephen Gottschalk, MD Member, St. Jude Faculty / Chair, Department of Bone Marrow Transplantation & Cellular Therapy / Endowed Chair in Bone Marrow Transplantation & Cellular Therapy

Terrence Geiger, MD, PhD Member, St. Jude Faculty / Senior Vice President and Deputy Director for Academic and Biomedical Operations / St. Jude Endowed Chair in Pediatrics

Elizabeth Fox, MD Member, St. Jude Faculty / Senior Vice President, Clinical Trials Research / Associate Director for Clinical Research, Comprehensive Cancer Center

Richard Finkel, MD Member, St. Jude Faculty / Director, Center for Experimental Neurotherapeutics / Endowed Chair in Neurotherapeutics

Frank Fazio, President of the Children's GMP & VP of Therapeutics, Prod

Liza Johnson, MD, Associate Member, St. Jude Faculty / Director, Bioethics Program