7:00 am Registration & Coffee
Reaffirming the Future of iPSC-Derived Cell Therapies to Benchmark the Road to Clinical Success
7:50 am Chair’s Opening Remarks
8:00 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
9:00 am Investigating 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
9:10 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 autologous 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
10:00 am Improved Anti-Tumor Immune Function of TGFβR2 Knock Out & IL-15 Knock In iPSCDerived 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
10:30 am Morning Refreshment Break & Speed Networking
DISCOVERY & TRANSLATION TRACK
Optimizing Gene Editing Protocols for Improved iPSC Product Safety
12:00 pm Overcoming Manual Bottlenecks to iPSC Reprogramming to Improve Efficacy & Efficiency
Synopsis
- Patient-specific induced pluripotent stem cells (iPSCs) are a valuable resource in the development of models for studying unique disease or drug responses
- Despite the potential of these cells, reprogramming has low efficiency (<1%), instability of pluripotency, and higher chance for mutations. Reprogramming is long, labor intensive and manual, and requires additional screening to derive a monoclonal population
- The CellRaft® Array offers a favorable culture environment for fragile iPSCs by providing flask-like culture conditions combined with single-cell segregation, and CellRaft AIR® Technology can accelerate reprogramming by improving efficiency, automation and cell viability
12:15 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
12:00 pm Suspension Culture of Autologous Human Induced Pluripotent Stem Cells in a Single- Use Vertical-Wheel® Bioreactor System Improves Stem Cell Quality, Quantity & Function
Synopsis
- Vertical-wheel® bioreactor system provides most optimal and scalable platform for human iPSC expansion
- Bioreactor-driven 3D iPSC culture enables prime to naive pluripotency transition
- Suspension iPSCs form more mature teratomas with defined tri-germ layer tissues
12:30 pm Promises & Challenges of Translating PSC-Derived Natural Killer Cells Manufacturing CMC Protocols
Synopsis
- Developing a Platform for a Scalable Solution For Off-The-Shelf NK Cell Therapies
- Safety Considerations of HebeCell’s PSC-NK
- NK-Specific Challenges and Solutions for CMC protocol
1:00 pm Lunch Break & Networking
DISCOVERY & TRANSLATION TRACK
Strengthening Preclinical Models to Improve Translation of Data to the Clinic
2:00 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
2:30 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
Learning the Landscape: Investors’ Perspectives on iPSC Therapies to Inform Successful Funding Proposals
2:00 pm Investors Deep Dive: Bridging the Gap Between Investors & Investigators to Establish a Mutual Understanding & Facilitate Meaningful Relationships
Synopsis
An introductory panel from leading investors in the cell therapy space uncovering the nonnegotiables and current perspectives on investing into iPSC-derived products. Rounding off with a group discussion on how to unify investor expectations with clinical reality
- 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
3:00 pm Afternoon Break
Discussing Data Requirements for Regulatory Approval of iPSC-Derived Cell Therapies
3:40 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
4:10 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