Plenary Session

PLENARY I: PRESIDENTIAL SYMPOSIUM (Grand Hall, L3)

Plenary Speaker(s)

• 

  Claire Booth, MBBS MSc PhD FRCPCH (she/her/hers)

  Infection, Immunity and Inflammation Department
  UCL Great Ormond Street Institute of Child Health, United Kingdom
• 

  Michael Pepper, MBChB, PhD, MD, PD (he/him/his)

  Medical Immunology
  University of Pretoria, South Africa
• 

  Masayo Takahashi, MD, PhD (she/her/hers)

  Vision Care Inc., and Kobe City Eye Hospital, Ritsumeikan University, Japan
• 

  Vidita Vaidya, PhD (she/her/hers)

  Biological Sciences
  Tata Institute of Fundamental Research, India

This session delves into groundbreaking advancements in gene therapies, focusing on the hematopoietic stem cell (HSC) system. Topics include gene addition and editing techniques, including CRISPR/Cas9 and prime editing, and their applications in treating genetic diseases affecting the blood, central nervous system (CNS), skeletal systems, and more. This session will also cover emerging in vivo gene therapy strategies targeting HSCs and immune cells, as well as the regulatory, accessibility, and economic challenges associated with these advanced therapies.

Presentations:

• THE PRESENT AND FUTURE OF GENE THERAPIES FOR INBORN ERRORS OF IMMUNITY
  

  Plenary Speaker: Claire Booth, MBBS MSc PhD FRCPCH (she/her/hers) – UCL Great Ormond Street Institute of Child Health
• MONOAMINES, MITOCHONDIA & MOOD
  

  Plenary Speaker: Vidita A. Vaidya, PhD (she/her/hers) – Tata Institute of Fundamental Research
• ENSURING GLOBAL ACCESS TO ADVANCED THERAPIES
  

  Plenary Speaker: Michael S. Pepper, MBChB, PhD, MD, PD (he/him/his) – University of Pretoria
• SUSTAINABLE RETINAL CELL THERAPY
  

  Plenary Speaker: Masayo Takahashi, MD, PhD (she/her/hers) – Vision Care Inc., and Kobe City Eye Hospital, Ritsumeikan University