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CDCM 8th Annual Symposium

The symposium is now over. Thank you to everyone that attended!

Join us on March 13, 2024 for the CDCM's 8th annual symposium and learn about new cell, gene, and regenerative medicine discoveries, pre-clinical development and clinical trials of curative medicines, and challenges with bringing these cures to patients to meet unmet medical needs. Exploration and discussion of these topics will be held at this one-day conference for clinicians, basic scientists, patient advocates, translational investigators, and biotech and pharmaceutical experts. More than 700 people are expected to attend, including a growing number of international participants from Europe, Asia Pacific, and the Middle East.

Each year, the CDCM hosts an annual symposium to engage in scientific discussion about new discoveries and developments in the cell and gene therapy field. The 8th annual symposium will host speakers to present on topics related to the field, including new cell, gene, and regenerative medicine discoveries; pre-clinical development and clinical trials of curative medicines; and challenges with bringing these cures to patients with unmet medical needs.

This year's host of renowned speakers include:

Lorenz Studer, MD
Mark A. Skylar-Scott, PhD
Fabrizia Urbinati, PhD
Michelle Monje, MD, PhD
Henry T. Greely, JD
Johannes van der Loo, PhD
Alessandro Aiuti, MD, PhD
Nicole Verdun, MD
Yun Sil Chang, MD, PhD

Additionally, the symposium will be highlighting 2 products that were developed by the CDCM and tell the story from the bench to bedside. Two panels will take place in the symposium, the IPEX Syndrome Treatment Program and the Fanconi Anemia Treatment Program, both of which were spearheaded at Stanford University.

Agenda

Program

Breakfast (7:30 AM)

Opening Remarks (8:00 - 8:30 AM)

Paul King, President and CEO, Stanford Children's Health

Lloyd Minor, MD, Dean, School of Medicine, Stanford Unviersity (Pre-recorded)

David Entwistle, President and CEO, Stanford Health Care (Pre-recorded)

Matthew Porteus, MD, PhD, Director, CDCM, Stanford University

Opening Keynote (8:30 - 9:10 AM)

Introduction to Keynote: Tony Oro, MD, PhD, Associate Director, CDCM, Stanford University

Keynote: Lorenz Studer, MD, Founding Director, Center for Stem Cell Biology, Memorial Sloan Kettering Cancer Center

“Developing a human PSC-based cell therapy for the treatment of Parkinson’s disease”

CDCM Translational Success From the Bench to the Bedside (9:10 - 10:25 AM)

Story of Treating IPEX Syndrome

Rosa Bacchetta, MD, Professor (Research) of Pediatrics - Stem Cell Transplantation, Stanford University

Prachi Wani, M.S., Senior Technical Manager for Process Development and Manufacturing, LCGM, Stanford University

Patient Perspective

15 Minute Discussion and Q&A

Break (10:25 - 10:45 AM)

From Discovery to Translation (10:45 - 12:00 PM)

(10:45 - 11:10 AM) ⸻ Mark A. Skylar-Scott, PhD, Assistant Professor, Stanford Bioengineering Basic Science and Engineering Initiative, Children's Heart Center, Stanford LPCH

“From the Petri Dish Towards Whole Organ Bioprinting”

(11:10 - 11:35 AM) ⸻ Fabrizia Urbinati, PhD, Director of Cell and Gene Therapies; Innovative Medicine Accelerator, Stanford University

"Translating Cell and Gene Therapies with the Stanford Innovative Medicines Accelerator"

(11:35 - 12:00 PM) ⸻ Michelle Monje, MD, PhD, Milan Gambhir Professor of Pediatric Neuro-Oncology, Department of Neurology and Neurological Sciences Investigator, Howard Hughes Medical Institute, Stanford University

"Cellular therapy for a lethal childhood brain and spinal cord cancer"

Lunch Break with Sponsor Presentations (12:00 - 1:00 PM)

Critical Partnerships in Cell Therapeutics: Ethics, Regulation, and Manufacturing (1:00 - 2:15 PM)

(1:00 - 1:25 PM) ⸻ Henry T. "Hank" Greely, JD, Deane F. and Kate Edelman Johnson Professor of Law; Director, Stanford Center for Law and the Biosciences

"The Ethics of Cell and Gene Therapy: The Current Moment"

(1:25 - 1:50 PM) ⸻ Nicole Verdun, MD, Super Office Director, Office of Therapeutic Products, Food and Drug Administration

“Cell and Gene Therapy Regulatory Updates” (Virtual Presentation)

(1:50 - 2:15 PM) ⸻ Johannes van der Loo, PhD, Director, Clinical Vector Core, Children's Hospital of Philadelphia; Adjunct Associate Professor, Dept. of Pathology and Laboratory Medicine, University of Pennsylvania

"Chasing industry expectations in academic viral vector manufacturing"

Break (2:15 - 2:40 PM)

CDCM Translational Success From the Bench to the Bedside (2:40 - 3:55 PM)

Story of Treating Fanconi Anemia

Agnieszka Czechowicz, MD, PhD, Lorry I. Lokey Faculty Scholar; Assistant Professor of Pediatrics; Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine; Stanford University School of Medicine

Rajni Agarwal, MD, Professor (University Medical Line) of Pediatrics - Stem Cell Transplantation, Stanford University

Isis Sroka, PhD, Chief Scientific Officer, Fanconi Anemia Research Fund

Patient Perspective

15 Minute Discussion and Q&A

International Keynote - Virtual Presentation (3:55 - 4:20 PM)

Introduction to Keynote: Tony Oro, MD, PhD, Associate Director, CDCM, Stanford University

International Keynote: Yun Sil Chang, MD, PhD, Professor of Pediatrics, Samsung Medical Center, Sunghyunkwan University School of Medicine

"Mesenchymal stem cell therapy for intractable neonatal brain disorders"

Closing Keynote (4:20 - 5:00 PM)

Introduction to Keynote: Maria Grazia Roncarolo, MD, Founding Director, CDCM, Stanford University

Keynote: Alessandro Aiuti, MD, PhD, Deputy Director, San Raffaele Telethon Institute for Gene Therapy (SR-Tiget); Professor of Pediatrics, Vita-Salute San Raffaele University, Milan, Italy

“Haematopoietic stem cell gene therapy for inborn errors of immunity and metabolism”

Closing Remarks (5:00 - 5:10 PM)