ROUNDTABLE Overcoming Barriers to iPSC Market Readiness: Part I
Wednesday, May 31, 2023 09:15 AM - 10:15 AM
Room 242A
Manufacturing Tools & Technology
Roundtable
What game changing technologies can overcome the barriers to iPSC scalability and manufacturing.
Moderator
Julie Allickson, PhD, Otto Bremer Trust Director, Biomanufacturing and Product Development, Center for Regenerative Biotherapeutics/Associate Professor of Regenerative Medicine, Michael S. and Mary Sue Shannon Director of Mayo Clinic's Center for Regenerative Biotherapeutics, USA
Panelists
Kapil Bharti, PhD, Senior Investigator, National Institutes of Health, USA
Maxime Feyeux, PhD, Co-founder and CSO, TreeFrog Therapeutics, France
Uma Lakshmipathy, PhD, Head Translational Sciences, Pharma Services , Thermo Fisher Scientific, USA
Timothy Nelson, MD, PhD, Director of the Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome , Mayo Clinic, USA
Session Description
Pluripotent stem cells are an appealing renewable source of cells for application in cellular therapies to treat diverse disease indications. A major consideration is to ensure safety, which is reliant on various aspects starting with careful selection of iPSCs to scalability and manufacturing of iPSC and iPSC-derived cells.
While the challenges are steep, steady progress has been made to employ a risk-based approach for its successful progression towards the clinic. The availability of clinical-grade raw materials and broad choice of analytical methods has enabled translation toward processes compliant with cGMP manufacturing albeit with a few challenges. The complex process of reprogramming and subsequent differentiation comprises of highly manual and open steps thus prone to contamination. Expansion and modification of iPSC enhances the risk of genome instability further compounded by the emerging regulatory guidance on the types of assays needed to establish safety. Together, these challenges are a barrier to scalable manufacturing required to achieve the required dose. Despite current gaps, iPSC-derived cells have been successfully progressed to Phase I clinical trials and notably used in the therapeutic intervention of iPSC derived RPE cells for AMD. This success has laid the foundation for developers designing clinical studies for other indications for both autologous and allogeneic approaches. This session will address advancements in technology, manufacturing solutions and clinical study designs with in the current regulatory framework.
Learning Objectives
Moderator
Julie Allickson, PhD, Otto Bremer Trust Director, Biomanufacturing and Product Development, Center for Regenerative Biotherapeutics/Associate Professor of Regenerative Medicine, Michael S. and Mary Sue Shannon Director of Mayo Clinic's Center for Regenerative Biotherapeutics, USA
Panelists
Kapil Bharti, PhD, Senior Investigator, National Institutes of Health, USA
Maxime Feyeux, PhD, Co-founder and CSO, TreeFrog Therapeutics, France
Uma Lakshmipathy, PhD, Head Translational Sciences, Pharma Services , Thermo Fisher Scientific, USA
Timothy Nelson, MD, PhD, Director of the Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome , Mayo Clinic, USA
Session Description
Pluripotent stem cells are an appealing renewable source of cells for application in cellular therapies to treat diverse disease indications. A major consideration is to ensure safety, which is reliant on various aspects starting with careful selection of iPSCs to scalability and manufacturing of iPSC and iPSC-derived cells.
While the challenges are steep, steady progress has been made to employ a risk-based approach for its successful progression towards the clinic. The availability of clinical-grade raw materials and broad choice of analytical methods has enabled translation toward processes compliant with cGMP manufacturing albeit with a few challenges. The complex process of reprogramming and subsequent differentiation comprises of highly manual and open steps thus prone to contamination. Expansion and modification of iPSC enhances the risk of genome instability further compounded by the emerging regulatory guidance on the types of assays needed to establish safety. Together, these challenges are a barrier to scalable manufacturing required to achieve the required dose. Despite current gaps, iPSC-derived cells have been successfully progressed to Phase I clinical trials and notably used in the therapeutic intervention of iPSC derived RPE cells for AMD. This success has laid the foundation for developers designing clinical studies for other indications for both autologous and allogeneic approaches. This session will address advancements in technology, manufacturing solutions and clinical study designs with in the current regulatory framework.
Learning Objectives
- Translation and barriers to iPSC scalability/manufacturability
- Risk-based approaches to regulatory compliant processes/analytics
- Consideration for clinical studies with autologous iPSC therapies and implications for allogeneic approaches
Sponsored by:
Julie Allickson
Director, Mayo Clinic Center for Regenerative Biotherapeutics Regenerative Biotherapeutics/Associate Professor of Regenerative Medicine
Michael S. and Mary Sue Shannon Director of Mayo Clinic's Center for Regenerative Biotherapeutics
Moderator
Director, Mayo Clinic Center for Regenerative Biotherapeutics Regenerative Biotherapeutics/Associate Professor of Regenerative Medicine
Michael S. and Mary Sue Shannon Director of Mayo Clinic's Center for Regenerative Biotherapeutics
Moderator
Timothy Nelson MD, PhD
Director of the Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome
Mayo Clinic
Panelist
Director of the Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome
Mayo Clinic
Panelist