7:30 am Registration & Coffee

8:30 am Chair’s Opening Remarks

Optimization of the Stem Cell Procedure to Generate Islet-like Structures

8:45 am Approaches to Functionally Mature Stem Cell-Derived Beta Cells For Therapy

  • Jeffrey Millman Assistant Professor – Medicine & Biomedical Engineering, Washington University St Louis

Synopsis

• State-of-the-art methodologies for generating stem cell-derived beta cells will be discussed
• Stem cell-derived beta cells are glucose-responsive and capable of reversing diabetes in mice
• Suspension culture for scalable cell production possible
• Functional parity with cadaveric human islets has been difficult to achieve

9:15 am High-Throughput Generation of Standardized Human iPSC Derived Pancreatic Beta Cell Islets

Synopsis

• We have established an automated pipeline for the optimization of iPSC-based differentiation protocols
• This pipeline is currently being applied to the differentiation of pancreatic beta cell islets from human iPSC
• Automation of beta islet differentiation allows parallel differentiation of multiple patient derived cells for population-based disease modeling and drug screening
• Protocol and process standardization also allows precise comparison of genetically modified pancreatic beta cells

9:45 am The Development of Allogeneic Mesenchymal Stem Cell Therapies for Type 1 Diabetes

Synopsis

  • Preserve endogenous insulin production in adult patients diagnosed with type I diabetes by immunomodulatory allogeneic mesenchymal stromal cells
  • Redundant source of primary hMSC for scalable manufacturing
  • Selection algorithm developed to select optimal cells based on multiple functional assays and mechanisms of action
  • Drug product currently in two clinical trials

10:15 am Preclinical Predictive Models: In Vitro Generation of Functionally Mature Beta-Cells from hiPSCs

  • Marine Kraus Team Leader, Stem Cells, Nestle Institute of Health Sciences

Synopsis

• hiPSCs can mature towards insulin producing cells in vivo
• hiPSCs can differentiate in vitro towards insulin secreting cells
• beta-like cells exhibit glucose regulated insulin secretion, displaying the first and second insulin release phases, characteristic of human islets

10:45 am Morning Refreshments & Networking

11:15 am Immunotherapy for Type 1 Diabetes

Synopsis

• Type 1 diabetes is an immune mediated disease. Over the past three decades there have been a number of clinical trials with immune interventions
• Trials have been directed at interdicting the type 1 diabetes disease process in an attempt to prevent the development or progression of the disease. Trials have also attempted to stabilize (and potentially even reverse) the disease. Most trials have either failed to have an effect or have had only a transient beneficial effect
• Newer trials are being designed in an attempt to better succeed at maintaining protracted beta cell function. Some trials use multiple agents directed at different components of the immune system
• Ideally, intervention would induce tolerance or permanently arrest the immune process. Recurrence of autoimmunity has been reported with beta cell replacement therapy in the absence of graft rejection. Thus, beta cell replacement therapies also will require control of the immune response directed against beta cells, and prevention of rejection

11:45 am Engineering Regulatory T Cells for Autoimmunity, Inflammation & Transplantation

  • Jason Fontenot Senior Vice President, Cell Therapy, Sangamo Therapeutics

Synopsis

• 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
• Engineering regulatory T cells beyond antigen recognition

12:15 pm Focusing on the Regulatory Considerations of Cell Therapies for Diabetes

12:45 pm Lunch & Networking

1:45 pm Panel Discussion: Moving Towards Commercialization – Forward Thinking

Synopsis

• Determining suitable risk analyses for supply chain, logistics and durability of suppliers
• Debating diversifying source providers to avoid supply deficits
• How do you ensure providers can meet the scale you need?

3:00 pm Chair’s Closing Remarks

3:05 pm Close of Summit