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Cell and Gene Therapy Approaches for Diabetes

Expert Speakers

From the advent of CRISPR technologies to the approval of CAR engineered technologies, gene engineering and editing is all the rage as these approaches show success in treating and curing patients.

As cell and gene therapies advance many off these innovations are being utilised in the journey to finding curative treatments for diabetes. From CAR-Tregs to targeting aberrant genetic pathways we explore the potential and opportunities that lie with gene engineering and editing, discussing:

  • Gene transfer methods
  • Cell vs. gene therapy approaches
  • Manufacturing considerations
  • Cost vs. value of each approach

As the field progresses and takes advantage of novel gene engineering/editing innovations, join leaders in development as they share key learnings in case studies, have an open and frank panel discussion and become an active participant in the conversation during breakout discussion groups.

Key Topics of Discussion

*All agenda times in EST

9:00 am Introduction & Opening Remarks

9:20 am CASE STUDY: Exploring Gene Editing as a Means to Protect Transplanted Cells from a Patients’ Immune System


• Learning how the advent of novel gene editing tools provides unique opportunities to disassemble MHC components in stem cell lines used to produce pancreatic-lineage cells to prevent presentation of allo-antigens and autoantigens by the stem cell-derived beta cells and recognition by memory autoimmune cells in T1D patients
• To what extent can eliminating MHC class I and class II expression reduce alloimmune and autoimmune-mediated rejection of beta cells?
• Discussing the importance of speed, specificity, and multiplexing efficiency of gene editing systems to maximize editing efficacy

9:40 am CASE STUDY: Engineering CAR Regulatory T Cells (CAR-Tregs) for Diabetes


• Engineering regulatory T cells with a Chimeric Antigen Receptor (CAR) to generate antigen specific Tregs
• CAR-Tregs as a new modality to restore immune tolerance
• Harnessing new potential activity of Tregs beyond their suppressive function to bring new medicine to patients

10:00 am CASE STUDY: Gene Therapy Approaches for Treating Diabetes


• Suppression of autoreactive T cells to prevent islet destruction prophylactically
• Strategies for the replacement of the insulin gene for curative therapeutics
• Gene transfer methods and gene therapy techniques for the maintenance of euglycemia in the treatment of T1D

10:20 am Speed Networking

11:00 am PANEL DISCUSSION: Advantages or Limitations of Cell and Gene Therapies


• Pros and cons of cells vs gene approaches
• Gene transfer methods: non-viral vs. viral
• Genetically engineering cells for enhanced efficacy
• Cost vs. value of each methodology
• What does the manufacturing strategy for these approaches look like?



• What are the possible therapeutic approaches of gene therapies for diabetes and how do they compare
with cell replacement approaches?
• How can we optimize cell therapy efficacy through utilizing gene engineering mechanisms?
• How can gene engineering be utilized to reduce alloimmune and autoimmune-mediated rejection of cells?
• What is the importance and impact of speed, specificity, and multiplexing efficiency of gene editing systems
to maximize editing efficacy?

12:45 pm Feedback Summary & Key Takeaways

1:00 pm End of Working Group C