4th Annual Robert J. Gorlin Symposium

The Situation Room: Gene Therapy in the Real World 

Thursday, February 6, 2025
17:45 – 19:00 PST

Overview:

Join colleagues for an intensive 75-minute panel discussion addressing the hottest controversies in gene therapy. Expert panelists—trailblazers with global perspectives—will focus on the contentious implications of the economic, regulatory, and ethical dimensions of single-administration (“one and done”) treatments. An engaging, dynamic exchange of often contrasting views will include:

  • Clinical Trials: Examining the significant financial and ethical considerations involved in funding clinical trials
  • Regulation: Analyzing complex approval processes and potential reforms.
  • Reimbursement: Exploring innovative payment models for gene therapies.
  • Accessibility: Addressing ethical considerations and healthcare equity.

Do not miss this consequential debate on the evolving gene therapy landscape and learn actionable steps that may help deliver gene therapies to the patients who need them. Register today.

Format:

  • This high-energy session will be presented as an expert panel discussion, similar to a news program expert discussion including targeted questions, discussion and open conversations. 
  • Prepared questions will be directed to individual panelists, and include group discussion, followed by audience Q&A to address the numerous controversies and challenges for gene therapy not only today, but also looking ahead into the future.

Course Director:
Jeanine R. Jarnes, PharmD, MSc, BCOP, BCPS
Assistant Professor, Department of Pediatrics
University of Minnesota Medical School
Pharmacotherapy for Inherited Metabolic Diseases
Advanced Therapies Department
College of Pharmacy, Experimental and Clinical Pharmacology
University of Minnesota
Minneapolis, MN, USA

Chair:
Philip J. (P.J.) Brooks, PhD
Deputy Director, Division of Rare Diseases Research Innovation
NIH – NCATS
Bethesda, Maryland, USA

Speakers:
James M. Wilson, MD, PhD
University of Pennsylvania
Gemma Biotherapeutics and Franklin Biolabs
Philadelphia, Pennsylvania, USA

Simon Jones, MBChB
Consultant in Paediatric Inherited Metabolic Disease
Senior Lecturer, The University of Manchester
Manchester, UK

Francis Pang
Vice President of Global Market Access
Orchard Therapeutics
London, UK

Emil Kakkis, MD, PhD
Chief Executive Officer, President and Director
Ultragenyx
Novato, California, USA

David Rind, MD, MSc
Chief Medical Officer
Institute for Clinical and Economic Review (ICER)
Boston, Massachusetts, USA

Thank you to the sponsors for this session

The Annual Robert J. Gorlin Symposium honors the work of Robert James Gorlin, DDS, PhD. Dr. Gorlin was a geneticist, maxillofacial pathologist, and academician at the University of Minnesota School of Dentistry. Spanning over 50 years, his groundbreaking research in genetic disorders of the head and neck revolutionized the understanding of the morphology of lysosomal diseases and many other genetic disorders.

View previous Robert J. Gorlin Symposium agendas and speakers.

Sponsorship opportunities are still available for this session. Click here for more information.