Please Note: All Timings Listed as EST

Day One

Wednesday, December 8 2021

9:00 am Online Registration & Virtual Coffee

9:15 am Chair’s Opening Remarks

Demonstrating Safety & Efficacy of iPSC-Derived Cells to Maximize Clinical Translation

9:30 am Industry Leaders’ Fireside Chat: Paving the Way For the Future of Cell Therapy with iPSCs

Synopsis

An executive panel discussion from the industry leaders of the field to set the scene on iPSC-derived
therapies. Ask your questions live to understand the expert’s thoughts on key topics including:

  • Exploring why iPSC’s should be selected as the choice of stem cell source compared to other sources such as donor derived MSCs or cord blood derived HSC’s
  • Understanding the differences in reprogramming efficiency to determine the best stem cell source
  • Are differences in cell sources reflected in meaningful functional differences?
  • Discussing rationale for differentiating to NK cells versus T cells

10:00 am A Platform for Developing and Characterizing Autologous iPSCs for Parkinson’s Disease

Synopsis

  • Using an AI-based genomics approach for manufacturing QC and rapid/ reproducible identification of specific cell types
  • Developing proprietary machine learning algorithms for predicting fate of iPSC derived cells
  • Optimizing surgical approaches that will improve cell delivery

10:30 am Navigating the Workflow From Process Development to GMP Manufacturing for Making Clonal iPSC-Derived Master Cell Banks

Synopsis

It is essential for clinical manufacturing to have a complete GMPcompliant
cell culture workflow for iPSC’s
• We will present data on optimising a range of factors that contribute to forming
GMP-compatible processes in the early stages of iPSC cell line development
• A GMP-compatible recombinant matrix, media and dissociation
reagents all contribute to this process and can provide optimal
conditions for single cell seeding
• We will also discuss some of the vendor and instrumentation
procurement considerations in planning for GMP cell line development
processes through to manufacturing

11:00 am Virtual Speed Networking

Synopsis

Reinventing the face-to-face networking in the virtual world. We will pair you up with fellow attendees to break the ice and make new and lasting connections!

Keynote Clinical Case Studies

11:30 am First Completed Clinical Trial With an Allogeneic iPSC-Derived Product: Cymerus™ MSCs for the Treatment of Acute GvHD

Synopsis

  • Establishing manufacturing and QC processes suitable for production of clinical grade iPSC-derived product
  • Securing first regulatory approval for a clinical trial with allogeneic iPSC-derived cells
  • Encouraging clinical outcomes in patients with acute steroid-resistant GvHD

12:00 pm Repairing Dying Retinal Cells using iPSC Derived Retinal Pigment Epithelium (RPE) Tissue

Synopsis

  • Discussing the advantaged of utilizing retinal replacement tissue from patient derived iPSCs
  • Using next generation sequencing approaches to genetically analyze the oncogenic potential of the iPSC derived RPE cells
  • Assessing the efficacy of transplantation of the iPSC derived RPE tissue for treating age related macular degeneration and restoring vision in patients
  • Establishing GMP protocols for development of a clinical grade product with reproducible results in clinical trial

12:30 pm Session Details to Be Confirmed

  • Lise Munsie Director, Process Development and Analytical Development, CCRM

12:40 pm Lunch & Networking

Advancing Clinical Trial Strategy for Successful Clinical Outcome

1:50 pm Session Details to Be Confirmed

2:00 pm Establishing ‘Universal’ iPSCs versus iPSC banks to Reach a Wider Patient Population

Synopsis

  • Discussing the potential of universal iPSCs compared to utilizing iPSC banks from selected HLA donors
  • Maximizing population coverage and boost interchangeability in-between genetically distant populations to limit the size of the cell bank required
  • Overcoming the challenge of immune rejections with genetically modified cells by artificially expressed HLA-E
  • Outlining the alternative use of an off-the-shelf for a more cost effective mass production strategy to supercharge movement into human clinical trials

2:30 pm Autologous iPSC-Derived Cell Replacement Therapy

Synopsis

  • Autologous vs. allogeneic cell products
  • Which is better for what disease?
  • Challenges of qualifying a cell product for each patient

3:00 pm Developing iPSC-Derived NK Cell Therapies Against Solid Tumors

  • Wei Lei CSO, Cytovia Therapeutics

Synopsis

  • Combining gene-editing and iPSC-NK technologies to overcome tumor microenvironment
  • Combining iPSC-NK and NKp46 NK engagers for solid tumors

3:30 pm Afternoon Break & Poster Session

Utilizing Innovative Gene Engineering for Enhanced Persistency of iPSC-Derived Products

4:00 pm Engineering iPSCs to Derive Immune Cells with Improved Functions

  • Dan Kaufman CSO & Professor, Shoreline Biosciences; UC- San Diego

Synopsis

  • Strategies to engineer iPSCs with novel receptors or genetic deletions as a key platform to produce NK cells and macrophages with improved anti-tumor activity
  • Describe how iPSC-derived NK cells and macrophages may work together to mediate improved anti-tumor activity

4:30 pm Panel Discussion: Is Gene Editing Truly the Key to Unlocking the Power of iPSC-Derived Cell Therapy Products?

  • Gregory Fiore President & CEO, Exacis Biotherapeutics
  • Dan Kaufman CSO & Professor, Shoreline Biosciences; UC- San Diego

Synopsis

  • Investigating the need for genetically removing self-antigens for development of a true allogeneic, universal iPSC immune product
  • Creating immunological space for iPSC derived immunotherapies by carrying out lymphodepletion
  • How to gene modify a universal graft without losing the function of the graft

5:00 pm Overcoming Major Challenges of Cell Therapy with iPSCs

Synopsis

The use of iPSC as starting cell type for therapy development allows us to overcome significant challenges in the following areas:

  • Gene editing and product design
  • Potency of final product
  • Manufacturing challenges
  • Quality requirements

5:30 pm Chairman’s Closing Remarks & Close of Day 1

Translating Causation To Treatment - Reviewing The Microbiome's Role In Drug Development