Explore the Agenda
7:30 am Registration & Light Breakfast
8:20 am Chair’s Opening Remarks
Delivering from Pipeline-to-Patient & Exploring Clinical Milestones to Demonstrate Safety, Scalability & Commercial Readiness
8:30 am Advancing Hypoimmune iPSC‑Derived Dopaminergic Progenitors (NCR201) Toward Clinical Translation in Parkinson’s Disease
- Sharing early safety and tolerability insights from Nuwacell’s NCR201 Phase I/II trial
- Highlighting long-term efficacy and biological activity from investigator-initiated trials
- Building scalable CMC foundations aligned with NCR201’s accelerated development route
9:00 am Advancing Autologous iPSC-Derived Dopaminergic Neurons Clinical Development
- Presenting safety and engraftment data from an iPSC-derived neuronal therapy in Parkinson’s disease
- Evaluating early clinical signals and defining meaningful endpoints for regenerative neurology
- Demonstrating manufacturing comparability and critical quality attribute control while scaling toward pivotal development
- Aligning CMC and regulatory strategy to support durability claims and patient follow-up
9:30 am Session Reserved for PBS Biotech
10:00 am Morning Break & Speed Networking
Meet a cross-section of peers from R&D, CMC, QC, and regulatory functions in a focused, high‑energy setting designed to spark new collaborations. This session gives you immediate access to the innovators shaping the iPSC field, helping you expand your network and build meaningful connections from the outset
Preclinical Track
Advancing Safe Genome Editing to Protect Line Stability, Potency & Long-Term Control
11:00 am Integrating AI-Driven Genetic Programming to Accelerate Discovery and Control of Cell Fate Pathways
- Using AI-guided genetic programming (e.g., generative models, virtual cell models) to rapidly identify high-efficiency differentiation routes and streamline traditional protocol development
- Applying predictive AI models to large-scale, multimodal cell-state datasets to forecast phenotype stability, lineage fidelity, and safety risks, enabling earlier de-risking of preclinical decisions
- Translating multiplexed screening and perturbation data into closed-loop, AI-optimized genetic programs that produce reproducible, scalable differentiation protocols and accelerate progression toward therapeutic applications
11:30 am Implementing Allogeneic iPSC Engineering Strategies to Develop the Next Generation of iPSC Therapies
- Extracting platform lessons from Intellia’s allogeneic iPSC engineering approach to support the development of allogeneic iPSC therapies in regenerative medicine
- Establishing reprogramming, editing, screening, and differentiation workflows to enable downstream applications
12:00 pm Reserved for Summit Partners
Process Development & CMC Track
Aligning Innovation & Manufacturing to Create Scalable & Effective iPSC-Based Therapeutics
11:00 am Session Reserved for Ajinomoto
11:30 am Roundtable: Implementing Functional QC Frameworks to Predict Potency & Reduce Line-to- Line Variability Early in Development
- Using functional assays to assess differentiation, proliferative potential and therapeutic relevance early, reducing downstream manufacturing and clinical failure risk
- Addressing the industry-wide gap in functional characterization defining what must be measured preclinically to ensure products are safe
- Building QC systems that de-risk variability across clones, donors, and maturation states, ensuring controlled, predictable behavior before entering GMP workflows
12:00 pm Session Reserved for NOVA Biomedical
12:10 pm Lunch
Private lunch hosted by Lonza, please inquire with info@hansonwade.com for more information
Strengthening Preclinical Decision‑Making Around Autologous vs Allogeneic Pathways & Donor Line Selection
1:10 pm De-Risking iPSC Cell Line Selection to Enable Clinical Progression & Regulatory Readiness
- Establishing practical criteria for selecting genomically stable and clinically fit iPSC lines suitable for FDA-bound programs
- Applying updated cell line selection strategies to reduce downstream CMC, comparability, and regulatory risk
- Locking in early decisions that prevent costly reselection, re-qualification, and delays to clinical timelines
1:40 pm Optimizing Donor & Starting‑Line Selection to Enhance iPSC Quality & Reduce Downstream Variability
- Building multi‑donor and multi‑clone assessment frameworks to prevent over‑reliance on a single source and reduce variability
- Identifying donor‑derived biases: how age, source material, and intrinsic biology predispose iPSCs to certain lineages
- Navigating preclinical ethical and regulatory constraints that can stall animal work or destabilize program timelines
Delivering Manufacturable Gene Edited iPSCs Through Smart Design & Analytics
1:10 pm Implementing Robust Cell‑Based Characterization Strategies to De‑Risk Gene Edited iPSC Manufacturing
- Designing fit‑for‑purpose cell‑based assays to confirm correct genetic editing, functional phenotype, and product consistency early in development
- Integrating analytical characterization across molecular, vector, and cell teams to support scalable, compliant iPSC manufacturing workflows
- Lessons learned from building a core analytics function within a large pharma setting, balancing innovation, robustness, and regulatory readiness
1:40 pm Implementing GMP‑Compatible Gene Editing Strategies to Enable Scalable iPSC‑Derived Cell Manufacturing
- Translating complex immune‑evasion gene‑editing strategies into manufacturable iPSC platforms, without committing to multiple costly master cell lines
- Designing flexible, staged CRISPR editing and evaluation workflows to balance product customization, speed, and control in GMP manufacturing
- Navigating real‑world constraints in gene edited iPSC manufacturing, including CRISPR selection, IP and licensing considerations, and CDMO readiness
2:10 pm Afternoon Break & Poster Session
Share your latest findings and contribute to the scientific conversation shaping the future of iPSC‑derived therapies. Submit your abstract to showcase cutting‑edge data, novel platforms, or emerging insights across preclinical development, CMC, QC, and clinical translation.
Turning iPSC Discovery into Demonstrable Clinical Impact, New Data & Real-World Insight Development Lessons
3:10 pm Session Reserved for Catalent
3:40 pm Advancing iPSC-Derived Ocular Cell Therapies to Improve Vision Restoration & Support Progression into Pivotal Trials
- Reviewing pipelines with iPSC-derived retinal cell replacement, focusing on preclinical engraftment, structural preservation, and early clinical functional readouts
- Defining meaningful visual endpoints, durability markers and safety criteria that demonstrate clinical benefit and support the move from early-phase studies toward larger efficacy trials
4:10 pm Accelerating Ocular Diseases Therapeutics: Gene Editing in iPSC-Derived Retinal Organoids
- Field overview of patient-derived iPSCs and retinal organoids, which provide a disease-relevant, human preclinical platform enabling functional validation of gene editing and cell therapies directly in photoreceptors, overcoming limitations of animal models
- Functional recovery validates therapeutic potential: restoration of key phototransduction components (e.g., PDE6, RHO, and others) in organoids confirms that genetic correction translates into biologically meaningful rescue, supporting singledose, gene and mutation-targeted therapies
- This approach helps to evaluate the efficacy, delivery strategies, and safety of genome-editing therapies across diverse monogenic diseases, supporting both regulatory decision-making and therapeutic development pipelines