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9:00 am Chair’s Opening Remarks

  • Tony Ho Head of Research & Development, CRISPR Therapeutics

Exploring Techniques to Protect Implanted Cells from a Patient’s Alloimmunity & Autoimmunity

9:15 am Developing Islet Cell Replacement Therapies for Type 1 Diabetes

Synopsis

• Manufacturing pluripotent stem cells and pancreatic progenitors
• Delivery systems to address immunity
• Preclinical and clinical progress

9:45 am The Beta-Cells’ Oxygenation Need: Moving from Theory to Practicality

Synopsis

• Why do we desperately need proactive supply of oxygen?
• What is the novelty/IP around it?
• What have we proved so far?
• What are the challenges and why do we believe we can overcome most of them?
• What is the plan moving forward?
• When do we expect to have the final verdict: Having the conclusions in our hands?

10:15 am Lessons Learned from Porcine Endocrine Xenotransplantation Studies in Immune Deficient and Immune Competent Recipients for the Treatment of Diabetes

Synopsis

• Advantages and challenges of a fetal porcine endocrine cell product in relation to encapsulation and immune suppression
• Functionality of porcine endocrine cells in immune deficient and immune competent recipients
• Possible immune protective strategies for fetal porcine endocrine cells

10:45 am Morning Refreshments & Speed Networking

11:45 am Examining Microencapsulation Methods for Implantation

Synopsis

• Introduction to microencapsulation technology and product pipeline
• Manufacturing process of encapsulated cells
• Unique attributes of encapsulation material
• Placement of capsules and mechanism of action
• Development of bio-artificial pancreas to treat Type 1 and insulin-dependent Type 2 diabetes by encapsulating genetically engineered liver cells, stem cells and beta islet cells

12:15 pm Defining New Combination Therapies to Intervene in Type 1 Diabetes

  • Matthias von Herrath Vice President & Senior Medical Officer, Type 1 Diabetes & Stem Cells, Novo Nordisk

Synopsis

  • The issues of immunity and reoccurrence of autoimmunity and allorecognition
  • Strategies using stem cells to overcome allorecognition
  • Discussing capsules and implantation

12:45 pm A Beta Cell Specific Gene Expression Methodology to Improve Islet Graft Function

Synopsis

  • Synthetic gene expression in beta cells as a strategy to regulate insulin release
  • Engineering functional pseudoislets from native islet endocrine cells
  • Pseudoislets transplanted into the anterior chamber of the mouse eye are properly vascularized and contribute to blood glucose homeostasis
  • In vivo functional imaging of beta cells within engrafted pseudoislets allows to evaluate the potential of this engineering approach in providing high-quality islets for clinical transplantation.

1:15 pm Lunch & Networking

2:15 pm Towards Manufacturing of Cells for Organ Engineering and Transplantation, Diabetes and Beyond

Synopsis

  • Cell culture manufacturing demands process understanding to attain product consistency, purity and function
  • Quality-by-Design principles are not commonly applied in living cell manufacture due to complexity of the products
  • High-Dimensional Design of Experiments (HD-DoE) is able to extract critical process parameters and offers as systems biology understanding of differentiation spaces, highly applicable to manufacturing of cells
  • Trailhead Biosystems is pioneering the application of HD-DoE for the generation of multiple specialized human cells
  • An HD-DoE based Development of a rapid, small molecule-only, method for the generation of dorsal pancreas and insulin producing cells will be shown

2:45 pm Functional Enrichment for Insulin Producing Cells from hPSC Large Scale Culture using Novel Cell Surface Marker Combinations

Synopsis

• Large scale production of hPSC-derived islets are composed from heterogeneous populations that include targeted therapeutic population (~40%) along with functionally irrelevant populations that include pancreatic progenitors and polyhormonal cells (~50%)
• Using cell-capture antibody array, novel antibodies to distinguish these populations were identified
• Utilizing MACS technology, large scale cultures were enriched using a novel antibody combination and re-aggregated
• In vitro and in vivo function is beneficial and superior to non-sorted cells
• Strategy to reduce the required cell mass loaded into an encapsulation device and potentially to decrease the device’s physical dimensions

3:15 pm Key Parameters for the Development of a Diabetes Cell Therapy Clinical Candidate

Synopsis

• Seraxis has developed a cGMP iPSC variant line from human pancreas that efficiently and preferentially re differentiates to the pancreatic lineage
• In combination with a tailored and differentiation protocol these cells generate highly pure clusters of hormone-secreting cells that mimic the function of mature human islets. This process is compatible with automated, closed-system cell manufacturing processes
• To enable survival and function within a host, Seraxis has developed a macroencapsulation device, SeraGraft, that is implanted to the abdomen and anchored to omental tissue. Seraxis’ mission is to rapidly and efficiently advance this therapy to the clinic

3:45 pm Afternoon Refreshments & Poster Session

4:15 pm Panel Discussion: Exploring Gene Editing as a Means to Protect Transplanted Cells from a Patients’ Immune System

Synopsis

• 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 alloantigens 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 autoimmunemediated rejection of beta cells?
• Discussing the importance of speed, specificity, and multiplexing efficiency of gene editing systems to maximize editing efficacy

5:15 pm Chair’s Closing Remarks

  • Tony Ho Head of Research & Development, CRISPR Therapeutics

5:30 pm End of Day 1