7:50 am Registration & Coffee
8:40 am Chair’s Opening Remarks
Reaffirming the Future of iPSC-Derived Cell Therapies to Benchmark the Road to Clinical Success
8:50 am iPSC Leaders Panel: Highlighting the Next Steps for the Future of iPSC-Derived Cell Therapies
Synopsis
- Reflecting on how far the field has come to reaffirm iPSCs as the cornerstone of regenerative cell therapies
- Understanding the prospects of iPSC and laying out the trajectory to unite industry experts to propel regulatory approval
- Discussing the progression of, and clinical readiness for, iPSC technologies to identify areas in need of greater attention
9:30 am A Phase 1/2a Study Using Autologous iPSC-Derived Retinal Pigment Epithelium for Treatment of Advanced Dry Age Related Macular Degeneration
Synopsis
- What is the biological need for RPE replacement as a potential therapy for AMD?
- How to translate an autologous iPSC-based therapy from bench-to-bedside?
- What are the future disease specific applications of a RPE-based cell therapy?
10:00 am Roundtable Discussion: To Auto or to Allo? Evaluating the Future of iPSC-Derived Cell Therapies to Inform Our Next Steps
Synopsis
- Considering the practicalities of a scaled-up autonomous approach to re-evaluate its clinical acceptance
- Understanding the transection between autologous and allogeneic translational approaches to promote a holistic iPSC strategy
- Identifying how industry, service providers and academia can collaborate to support the advancement of iPSCs to the clinic
10:30 am Investigated Stem Cell Genetic Integrity Using NGS Testing: Turning a Deep Analysis Into a Straightforward Interpretation
Synopsis
- Introducing the latest Next-Generation Sequencing (NGS) genomic stability assay range
- Enabling the detection of copy number variations (CNVs) such as the 20q amplicon and single nucleotide variations (SNVs) in genes such as TP 53 and BCOR amongst many more.
- Providing an end-to-end workflow solution that includes personalized bioinformatics analyses for comprehensive interpretation of the obtained data.
10:40 am Morning Refreshment Break & Speed Networking
DISCOVERY & TRANSLATION TRACK
Optimizing Gene Editing Protocols for Improved iPSC Product Safety
11:40 am Improved Anti-Tumor Immune Function of TGFβR2 Knock Out and IL-15 Knock In iPSC-Derived NK (iNK) Cells by TALEN® Editing
Synopsis
- The off-target effect of TALEN pairs were detected, and these TALEN®-edited iPSCs kept their pluripotency, exhibited normal morphology and karyotype.
- Engineered iNK cells with IL-15 KI and TGFβR2 KO consist of a homogeneous population of CD56+ NK cells (>99%) with the expression of several typical NK markers.
- The iNK cells with TGFβR2 KO are resistant to TGFβ-mediated suppression of activating receptors and conferred resistance to TGFβ-mediated suppression of cytotoxicity against HCC tumor cells.
12:10 pm Talk by our Innovation Partner, Cell Microsystems
12:20 pm Panel Discussion: Benchmarking Safety Considerations During Gene Editing Protocols to Ease Regulatory Burdens
Synopsis
- Balancing therapeutic potential of immortalized cells with safety considerations
- Discussing best practices for combatting offsite mutations to reduce adverse effects
- Monitoring cell behavior to prevent product contamination
REGULATORY & EARLY CLINICAL DEVELOPMENT TRACK
Refining Manufacturing Strategies to Increase Efficiency in CMC Protocols
11:40 am Developing a Scalable Process Using a Quality by Design Approach for High Yield Bioprocessing of Cell Therapy Products in Stirred Suspension-Based Bioreactor
Synopsis
- Scalable and controllable manufacturing of pluripotent stem cells with iterative process development
- Aligning cell manufacturing with QbD
- Process optimization and characterization using bioprocess solutions including automated feeding and in-process monitoring.
12:10 pm Talk by our Program Partner, PBS Biotech
12:40 pm Exploring Technologies to Enable Population-Wide Personalized iPSC Production
Synopsis
- Reaffirming the fundamental biology underpinning iPSCs to enable personalized medicine and therapy
- Benchmarking manufacturing challenges preventing the commercial development of iPSCs based personalized medicine and therapy
- Discuss technologies and innovation to enable population-wide personalized iPSC production
1:10 pm Lunch Break & Networking
DISCOVERY & TRANSLATION TRACK
Strengthening Preclinical Models to Improve Translation of Data to the Clinic
2:10 pm Tailoring the Preclinical Approach for Effective Translation of a Cell (-Derived) Therapy for Ischemic Stroke
Synopsis
- Aligning in vitro potency with relevant disease biology to inform potential therapeutic efficacy
- Using in vivo models to understand facets of translation that lead to improved efficacy
- Overcoming challenges in clinical development through better understanding of optimal delivery
2:40 pm Roundtable Discussion: Combatting Limitations with Preclinical Models to Improve Translation of iPSC-Derived Therapies to the Clinic
Synopsis
- Acknowledging current limitations with existing animal models to identify areas for greater discovery
- Discussing alternative approaches to enhance quality of preclinical efficacy, safety and toxicology data
- Benchmarking critical qualities of preclinical models to ensure accurate reflection of disease indications
REGULATORY & EARLY CLINICAL DEVELOPMENT TRACK
Discussing Data Requirements for Regulatory Approval of iPSC-Derived Cell Therapies
2:10 pm Determining Stability of Genome Editing Components to Streamline Regulatory Strategies
Synopsis
- Identifying critical components – the role of the ribonucleoprotein complex
- In-silico estimation of intra- and extra-cellular dilution of residuals
- In-vitro and ex-vivo stability of the ribonuclease complex and individual CRISPR components
2:40 pm Roundtable Discussion: Outlining High-Quality Efficacy & Potency Data to Boost Regulatory Approval
Synopsis
- Optimizing collection of efficacy data in a multidose process to create a strong data package for regulatory bodies in a manageable time frame
- Highlighting the importance of early consideration of potency assays and reference materials to accelerate phase II approval and increase confidence in clinical processes
- Considering characteristics that are fundamental in animal models to boost agency approval
3:15 pm Afternoon Break & Networking
Tackling In Vivo Survivability to Improve Clinical Tolerane
3:45 pm Exploring Delivery & Formulation Challenges of iPSC-Derived Cell Therapies to Improve In Vivo Survivability & Clinical Success
Synopsis
- Benchmarking indication specific challenges of cell therapy delivery to improve efficacy
- Enhancing engraftment and immune-protection to improve in vivo survivability
- Understanding dose control considerations and optimizing routes of administration to improve clinical trial outcomes
Strengthening Preclinical Models to Improve Translation of Data to the Clinic
4:15 pm Roundtable Discussion: Re-Evaluating Necessary Genetic Knock-Outs to Reduce Immune Burdens
Synopsis
- Reviewing current evidence determining the necessity of immune knock-outs to elevate regulatory approval
- Comparing persistency of cells in immune knock-out models to non-knock out models to assess essential edits
- Outlining data requirements to justify knock-out selection to unite the iPSC space
Learning the Landscape: Investors’ Perspectives on iPSC Therapies to Inform Successful Funding Proposals
3:45 pm Early Stage Venture Investing in iPSC Technology
Synopsis
- Why do we think this space is so important to fund?
- What type of advancements in technology do we, as early-stage investors, want to see?
- How can you as a company/syndicate investor work with NYBC Ventures to build/fund gamechanging companies?
Discussing Data Requirements for Regulatory Approval of iPSC-Derived Cell Therapies
4:15 pm Panel: Bridging the Gap Between Investors and Investigators to Establish a Mutual Understanding and Facilitate Meaningful Relationships
Synopsis
- Developing mutual agreements and understandings of the future of iPSCs to inform smart investments
- Analyzing costs of clinical plans to provide insight into how to lower costs and reduce investment burdens
- Mapping out plans to produce iPSC therapies faster and cheaper to increase investment excitement