Walter E. Washington Convention Center

Jun 15, 2025 7:15 AM - Jun 15, 2025 5:45 PM

801 Allen Y. Lew Place, NW, Washington, DC 20001-3614, USA

Cell and Gene Therapy Summit

Join multi-stakeholder dialogue, identifying the barriers to progress and charting the best path forward in global regulatory harmonization for cell and gene therapies.

Session 1: An Overview of the Evolution and Impact of Cell and Gene Therapy

Session Chair(s)

Representative Invited

Representative Invited

Irish Dental Association, Ireland

Genome editing is perhaps one of the most significant advancements in biomedical sciences over the past two to three decades. Since scientists' first attempts at genome editing in the 1970s and 1980s, the field has rapidly evolved, particularly with the introduction of CRISPR in 2009 — a more agile and cost-effective technology compared to earlier genome editing methods. These advancements have revolutionized medicine by offering pioneering potential treatment for genetic disorders, rare diseases, and certain types of cancer. With the development of novel delivery systems, advancements in manufacturing scalability, and next-generation gene editing tools, the field of cell and gene therapy (CGT) has helped shift experimental treatments to real-world clinical applications. However, challenges persist in these therapies, including safety concerns, ethical considerations, regulatory complexities and commercialization hurdles.

This session will overview the history of CGT and highlight the scientific breakthroughs that were made along the way amid variable regulatory landscapes and summarize the opportunity for impact on patients in many different therapeutic areas, including oncology, rare disease, Type 1 diabetes, etc. Attendees will be encouraged to contemplate on the future of CGTs and their impact on patient care.

Learning Objective :
  • Describe the role of CGTs in treating genetic disorders, rare diseases, and cancers
  • Learn about how different regulatory landscapes have shaped CGT adoption and current challenges in CGT development
  • Summarize the evolution of genome editing and its impact on CGTs

Speaker(s)

John  Tisdale

Keynote Speaker

John Tisdale

National Institutes of Health (NIH) , United States

Senior Investigator, Cellular and Molecular Therapeutics, NHLBI

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