7:30-8:00am

Breakfast

8:00-8:10am 

Opening Remarks: Dean Lloyd Minor, School of Medicine, Paul King, President and CEO, Lucile Packard Children's Hospital and David Entwistle, President and CEO, Stanford Health Care 

8:10-8:15am

Welcome: Matthew Porteus, MD, PhD, Associate Director, CDCM

8:15-9:00am

Introduction of Keynote: Irving Weissman, MD, Director, Institute of Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine

Opening Keynote: David Baltimore, PhD, President Emeritus, Robert Andrews Millikan Professor of Biology, California Institute of Technology

TITLE: “Therapy Enters the Genetic Era”

9:00-9:30am

Calvin Kuo, MD, PhD, Maureen Lyles D’Ambrogio Professor, Stanford University

TITLE: “Using organoids to model immune-mediated diseases”

9:30-10:00am 

Tom Wandless, PhD, Professor of Chemical and Systems Biology and, by courtesy, of Chemistry, Stanford University

TITLE: "Regulating Protein Stability with Ambitions for Cell Therapy"

10:00-10:30am

Kyle Loh, PhD, Assistant Professor of Developmental Biology (Stem Cell), Stanford University

TITLE: “A developmental roadmap to efficiently generate human tissue progenitors from pluripotent stem cells”

10:30-10:45am

BREAK

10:45-11:15am

Robbie Majzner, MD, Instructor in the Division of Pediatric Hematology-Oncology, Stanford University

TITLE: “Adoptive immunotherapy for pediatric solid tumors”

11:15-11:45am 

Maria Grazia Roncarolo, MD, Director, CDCM/Rosa Bacchetta, MD, Associate Professor of Pediatrics-Stem Cell Transplantation, Stanford University

TITLE: "Regulatory T-cell therapy for genetic and acquired immune-mediated diseases"

11:45-12:15pm

Natalia Gomez-Ospina, PhD, MD, Assistant Professor, Pediatrics, Genetics, Stanford University

TITLE: “Genome-edited human hematopoietic stem cells correct neurometabolic disorders: Proof-of-concept and safety studies for Mucopolysaccharidosis type I and Gaucher disease.”

12:15-1:00pm

LUNCH

1:00-1:30pm

Chaitan Khosla, PhD, Director, ChEM-H, Wells H. Rauser and Harold M. Petiprin Professor in the School of Engineering and Professor of Chemistry and, by courtesy, of Biochemistry

TITLE: “Bench-to-bedside Translation of Innovative Molecules at Stanford”

1:30-2:00pm 

Alice Bertaina, MD, PhD, Associate Professor of Pediatrics-Stem Cell Transplantation, Stanford University 

TITLE: “Alpha/beta T-cell depleted haploidentical stem cell transplantation: a new platform for adoptive immunotherapy”

2:00-2:30pm

John Day, MD, PhD, Professor, Neurology & Neurological Sciences Director, Stanford Neuromuscular Disorders Program, Stanford University

TITLE: “SMA – a prototype for neurodegenerative disorders”

2:30-2:45pm

BREAK

2:45-3:15pm

Peter Marks, MD, PhD, Director of the Center for Biologics Evaluation and Research (CBER) at the FDA

TITLE: “Optimizing the Clinical Development of Advanced Therapy Medicinal Products”

3:15-3:45pm

Fraser Wright, PhD, Principal, Wright Biologics Consulting

TITLE: “Gene Therapy Product Development: Technology Strategies for Curative

Precision Medicines”

3:45-4:15pm 

Lynn Frohnmayer, Fanconi Anemia Research Fund

TITLE: "Making Something from Nothing: One Family's Journey"

4:15-5:00pm

Closing Keynote: David Chang, MD, PhD, President, Chief Executive Officer and Co-Founder, Allogene Therapeutics

TITLE:  "Shifting from Autologous to Allogeneic CAR T Therapy”

5:00-5:05pm

Closing Remarks: Maria Grazia Roncarolo, MD, Director, CDCM 

5:05-6:00pm

WINE & CHEESE RECEPTION