7:30 am Registration & Coffee
8:20 am Chair’s Opening Remarks
Reflecting on iPSC Successes & Learnings to Understand the Prospects of iPSC-Derived Cell Therapies
8:30 am A Case Study to Benchmark the Road to Success For a Precision Engineered iPSC-Derived Effector Cell
Synopsis
- Experiences, learnings and challenges when moving to a PH1 clinical trial
- Laying the regulatory foundations
- Future directions with next-generation programs
9:00 am New Era of Heart Failure Treatment: Regenerative Cell Therapy Using Ventricular Specific Purified Cardiomyocytes
Synopsis
- Scalable method to eliminate undifferentiated iPSCs by difference of metabolism between iPSCs and cardiomyocytes
- Clinical data from FIH study of purified cardiomyocyte spheroids
- Platform technology to develop high quality iPSC-derived product
9:30 am An Automated Platform to Scale Autologous iPSC Therapy Production
Synopsis
- Developing automated reprogramming processes using a multidisciplinary automated workcell
- Designing & building automated closed clinical-grade systems for scalable autologous iPSC manufacturing
- Using image-based analysis and laser removal of cells to automate cell processes & reduce operator variability for massive scale
10:00 am Morning Refreshment Break & Networking
Diving Into Differentiation to Optimize iPSC-Derived Cell Functions & Fulfill Clinical Potential
11:00 am Precise and Mb-scale iPSC genome engineering for cell therapy & regenerative medicine by Logomix Geno-WritingTM Platform
Synopsis
- Geno-Writing™, an innovative genome-writing platform, facilitates precise and Mb-scale manipulation of genomic sequences within human ES/iPS cells.
- Universal donor iPSC lines were constructed through large scale deletion encompassing all of the HLA class I and the HLA class II gene loci, complemented with targeted integration of HLA genes of strategic choice.
- Through site-specific integration of ~40 kb gene cluster constructs, Geno-Writing™ enables creation of a library of iPSCs containing distinct combinations of multiple transgenes and functional screening of their derivatives.
11:30 am Directing Differentiation to High-Purity Cell Populations Appropriate for Cellular Therapy
Synopsis
- Examining current and novel technologies to successfully characterize undifferentiated cells to reduce product contamination
- Diving beyond karyotypic screening to drive reliable assessment of genomic integrity
- Evaluating the role maturity plays in differentiation to understand why iPSCs do not always fully differentiate into their potential
Improving Data Strategies to Streamline Clinical Success
12:00 pm GMP Process for High-Quality iPSC Manufacturing & Development of Improved Differentiation Methodologies
Synopsis
• Principle of reprogramming and key considerations of iPSC derivation and banking
• GMP-compliant iPSC manufacturing and improved iPSC differentiation protocols for cell therapy
12:30 pm Roundtable Discussion: Considerations of Quality Control Strategies to Increase Confidence in Preclinical Processes & Advance Candidates to the Clinic
Synopsis
• What reference and control materials should we be using for our QC and potency assays?
• Best practices for defining your endpoints for QC/potency assessments to highlight the effect on end products
• Identifying ways QC and the manufacturing process can be commonly optimized to increase regulatory acceptance
1:00 pm Lunch Break & Networking
The Future is iPSCs: Re-Evaluating Translational & Strategical Priorities to Drive Next Steps Across the iPSC Industry
2:00 pm Genetic Engineering of Allogeneic Donor Cells for Acceptance by the Host’s Immune System
Synopsis
- Overcoming the immune barrier in allogeneic cell transplantation – discussion of the concept of immune evasion
- Understanding the immune response to different iPSC-derived cell types in different transplantation sites
- From mice to patients – various preclinical and translational models to bridge the gap from bench to bedside
2:30 pm Diabetes cell therapy with Evotec iPSC-derived islet-like clusters in the Sernova Cell PouchTM
Synopsis
- Update on Evotec’s end-to-end integrated platform for iPSC-based cell therapeutics
- Results from pre-clinical testing of iPSC-derived islet-like clusters combined with the implantable Cell Pouch
2:40 pm Discovering How to Make iPSC Cell-Derived Therapies a Reality: A Pharma Perspective
Synopsis
- Benchmarking pharma priorities when translating a smaller scale process to a larger scale
- Streamlining the process to clinical success
- Highlighting common challenges and solutions of iPSC platform strategies to turbocharge iPSC therapies to the clinic
3:10 pm Afternoon Break & Networking
3:40 pm Exploring the Potential of Human Trophoblast Stem Cells (TSCs)
Synopsis
- Human trophoblast stem cells as a starting stem cell source with product-market fit
- GMP hTSC and hTSC-derived iPSC source with complete regulatory support package
- Out-licensing to save cell and gene therapy developers 3 to 5 years in time and $10M in costs
4:10 pm Evidence-Based Quantitative Decision Making in Cell Therapy Clinical Development
Synopsis
- Developing an evidence-based quantitative decision framework
- Benchmarking endpoints and data package development for decision making
- Discussing criteria for a go/no-go decision based on pre-specified target and/or reference values