Wednesday, May 29
08:00 AM - 08:30 AM

Welcome and Regulatory Roundup

Speakers:

Karen Nichols, Esq., VP, Regulatory and Quality, Magenta Therapeutics, United States

Dominic Wall, PhD, FFSc(RCPA), Director Pathology, Operations, Peter MacCallum Cancer Centre, Australia

Scott Burger, MD, Principal, Advanced Cell & Gene Therapy, USA
Speakers: Karen Nichols, Esq., VP, Regulatory and Quality, Magenta Therapeutics, United States Dominic Wall, PhD, FFSc(RCPA), Director Pathology, Operations, Peter MacCallum Cancer Centre, Australia...
08:30 AM - 09:30 AM

Global Regulatory Perspectives (GRP) Workshop Session 1 - CMC Considerations (including testing) for Pediatric Treatments

Moderator: 

Rebecca Lim, PhD, Associate Professor, Obstetrics & Gynaecology, Monash University, Australia

Confirmed Speakers: 

Ieva Ozolins, MD, Medical Advisor, Therapeutic Goods Administration, Australia
Yoshiaki Maruyama, PhD, Review Director, PMDA, Japan
Mo Heidaran, PhD, VP Technical, PAREXEL, United States
Moderator:  Rebecca Lim, PhD, Associate Professor, Obstetrics & Gynaecology, Monash University, Australia Confirmed Speakers:  Ieva Ozolins, MD, Medical Advisor, Therapeutic Goods Administration, A...
08:30 AM - 10:00 AM

Cell Processing Track Session 1 - Collection of Starting Product Before Cell Processing and Production

Co-Chairs:

Mara Neal, SH(ASCP)MT, MS, MBA, Cook Regentec, USA
Vicki Antonenas, BSc, MSc, Westmead Hospital, Australia

Speakers:

Needle to Needle of Cellular Therapy Products- An Introduction
Kenneth Micklethwaite, MBBS, PhD, FRACP, FRCPA, Westmead Hospital, Australia

Collection of Starting Product Before Cell Processing and Production
Sharlea Disney, Peter MacCallum Cancer Centre, Australia

Cell Collection and Preparation for iPSC Derivation Process
Wen Bo Wang, PhD, Fate Therapeutics, USA
Co-Chairs: Mara Neal, SH(ASCP)MT, MS, MBA, Cook Regentec, USA Vicki Antonenas, BSc, MSc, Westmead Hospital, Australia Speakers: Needle to Needle of Cellular Therapy Products- An Introduction Kennet...
08:30 AM - 10:00 AM

ISCT-CBA Cord Blood Series in Partnership with ASBMT Session 1 - Cord Blood Expansion

Chair:
Joanne Kurtzberg, MD, Duke University Medical Center, United States

Speakers:

Notch-mediated ex vivo expansion and directed differentiation of cord blood CD34+ cells for clinical application
Colleen Delaney, MD, MSc Nohla Therapeutics, United States

NiCord, an expanded cord blood product, accelerates engraftment after myeloablative conditioning 
Joanne Kurtzberg, MD, Duke University Medical Center, United States

Rapid and Robust CD4+ and CD8+ T-, NK-, B- and Monocyte Cell Reconstitution after Nicotinamide-Expanded Cord Blood Transplantation
Jaap Jan Boelens, MD, PhD, Memorial Sloan Kettering Cancer Center, United States
Chair: Joanne Kurtzberg, MD, Duke University Medical Center, United States Speakers: Notch-mediated ex vivo expansion and directed differentiation of cord blood CD34+ cells for clinical application ...
08:30 AM - 10:00 AM

MSC Workshop Session 1 - Clinical Trials Part I: MSC Sources, Applications and 'Off the Shelf' Products

Chair: Yufang Shi, PhD, Institutes for Translational Medicine, China

Speakers:
Commercial Scale Manufacturing Using Next Generation Technologies to Meet Peak Market Demand for Approved Cell Therapies
Paul Simmons, PhD, Mesoblast, Australia

iPSC-Derived Mesenchymal and Vascular Progenitor: A Novel Technology Platform for Regenerative Medicine
Igor Slukvin,MD, PhD, University of Wisconsin, United States

Clinical Development of MultiStem®, an Adult Stem Cell Therapy, for Treatment of Acute Injuries of the Central Nervous System
Robert W. Mays,PhD, Athersys Inc., United States
Chair: Yufang Shi, PhD, Institutes for Translational Medicine, China Speakers: Commercial Scale Manufacturing Using Next Generation Technologies to Meet Peak Market Demand for Approved Cell Therapies...
08:30 AM - 10:00 AM

ISCT-FACT Quality Bootcamp Session 1 - Quality Plans and Document Control

Confirmed Speakers:

Welcome
Phillip Johnson, MMEdSc(Path), Production Manager, Queensland Cord Blood Bank at the Mater, Australia

Incorporating IECs into your quality plan and written agreements
Patrick Hanley, PhD, Director of GMP for Immunotherapy, Children's National Health System Blood and Marrow Transplant Program, United States

Document Control
Guy Klamer, PhD, Cord Blood Bank Director, Sydney Cord Blood Bank, Australia
Confirmed Speakers: Welcome Phillip Johnson, MMEdSc(Path), Production Manager, Queensland Cord Blood Bank at the Mater, Australia Incorporating IECs into your quality plan and written agreements Pat...
09:30 AM - 10:00 AM

Global Regulatory Perspectives Case Study I

Panel Discussion: 

Rebecca Lim, Ieva Ozolins, Yoshiaki Maruyama, Mo Heidaran
Panel Discussion:  Rebecca Lim, Ieva Ozolins, Yoshiaki Maruyama, Mo Heidaran
10:00 AM - 10:15 AM

10:15 AM - 11:45 AM

Cell Processing Track Session 2 - Specialized Cell Processing

Co-Chairs:

Ruud Hulspas, PhD, Cellular Technologies Bioconsulting, Netherlands
Paul Wallace, PhD, Roswell Park Comprehensive Cancer Center, United States

Speakers:

Upstream Optimization to Improve Downstream Outcomes - Raw Material Selection Impacting Product Performance
Leela L Paris, PhD, Cook Regentec, United States

From Little Things… Scale Up for Cell Therapies
Frances Harding, PhD, Cell Therapies Pty, Australia

On-Site Academic Production of CAR T Cells- Challenges and Logistics
Paul Eldridge, PhD, UNC Lineberger Advanced Cellular Therapeutics Facility, USA
Co-Chairs: Ruud Hulspas, PhD, Cellular Technologies Bioconsulting, Netherlands Paul Wallace, PhD, Roswell Park Comprehensive Cancer Center, United States Speakers: Upstream Optimization to Improve ...
10:15 AM - 11:45 AM

Global Regulatory Perspectives (GRP) Workshop Session 2 - Risk Assessment for Administration of Non-conforming Products

Moderator:

David Ritchie, Deputy Director, Head, Bone Marrow Transplant Service, Australia
Presenters: 

Steven Oh, PhD, Deputy Director, Office of Tissues and Advanced Therapies, FDA, United States

So-Young Lee, PhD, Scientific Reviewer, Cell and Gene Therapy Products Division, National Institute of Food and Drug Safety Evaluation (NIFDS), Ministry of Food and Drug Safety (MFDS), Korea

Stephan Kadauke, MD, PhD, Assistant Director, Cell and Gene Therapy Lab, CHOP, United States
Moderator: David Ritchie, Deputy Director, Head, Bone Marrow Transplant Service, Australia Presenters:  Steven Oh, PhD, Deputy Director, Office of Tissues and Advanced Therapies, FDA, United States ...
10:15 AM - 11:45 AM

ISCT-CBA Cord Blood Series in Partnership with ASTCT Session 2 - Cord Blood for Neurological Disorders

Chair: 
Joanne Kurtzberg, MD, Duke University School of Medicine, United States

Speakers:

Cord blood based therapies for neurological diseases
Joanne Kurtzberg, MD, Duke University School of Medicine, United States

Human umbilical cord blood derived mesenchymal stem cells: Magic cure for neonatal brain injuries?
Won Soon Park, MD, PhD, Samsung Medical Center, Korea

Efficacy of allogeneic cord blood cell therapy combined with erythropoietin for children with cerebral palsy
MinYoung Kim, MD, PhD, CHA University, Korea
Chair:  Joanne Kurtzberg, MD, Duke University School of Medicine, United States Speakers: Cord blood based therapies for neurological diseases Joanne Kurtzberg, MD, Duke University School of Medicin...
10:15 AM - 11:45 AM

MSC Workshop Session 2 - Clinical Trials Part II: Asia Pacific Spotlight

Chair: Donald G. Phinney, PhD, Scripps Research Institute, United States

Speakers:

Therapeutic Potential of Bone Marrow-derived, pooled, Allogeneic Mesenchymal Stromal Cells (Stempeucel®) for Patients with  Critical Limb Ischemia due to Buerger’s disease
Pawan Kumar Gupta, MBBS, MD, DNB, PhD, Stempeutics Research, India

From bench to bedside --- the clinical study of mesenchymal stem/stromal cell therapy for acute inflammatory response syndrome
Andy Peng Xiang, PhD, Sun Yat-Sen University, China

Treatment of Spinocerebellar Ataxia with Allogeneic mesenchymal Stem Cells
Oscar K. Lee, MD, PhD, National Yang Ming University, Taiwan
Chair: Donald G. Phinney, PhD, Scripps Research Institute, United States Speakers: Therapeutic Potential of Bone Marrow-derived, pooled, Allogeneic Mesenchymal Stromal Cells (Stempeucel®) for Patien...
10:15 AM - 11:45 AM

ISCT-FACT Quality Bootcamp Session 2 - Performing Risk Assessment, What Now?

Confirmed Speakers: 

Phillip Johnson, MMEdSc(Path), Production Manager, Queensland Cord Blood Bank at the Mater, Australia
Confirmed Speakers:  Phillip Johnson, MMEdSc(Path), Production Manager, Queensland Cord Blood Bank at the Mater, Australia
Speakers
  • Phillip Johnson MMedSc (Path), Production Manager, Queensland Cord Blood Bank At The Mater (Speaker)
10:15 AM - 11:45 AM

Stem Cell Crystal Ball - Navigating Contemporary Practice and Imagining the Future

This session is focused towards practicing health professionals, health professionals in training, and those with a general interest in stem cell and gene therapy for human health. Attendees will be introduced to the basics of stem cell biology, including terminology and current evidence-based clinical applications of stem cells. Using clinical case studies as examples, we will explore recent breakthroughs and future directions in the transforming use of stem cells as cellular therapies in a range of diseases. Ethical, social and legal implications will be discussed in the context of responsibility and professional standards.

Speakers:

Getting a handle on stem cell biology and gene therapy
Ashley Ng, MBBS, BMedSci FRACP FRCPA PhD, Royal Melbourne Hospital and the Walter and Eliza Hall Institute of Medical Research, Australia

Uses in Contemporary Medical Practice
Rosa McCarty, PhD GCUT, University of Melbourne, Australia

Issues beyond the lab
Ashley Ng, MBBS, BMedSci FRACP FRCPA PhD, Royal Melbourne Hospital and the Walter and Eliza Hall Institute of Medical Research, Australia
This session is focused towards practicing health professionals, health professionals in training, and those with a general interest in stem cell and gene therapy for human health. Attendees will be i...
11:45 AM - 01:00 PM

Lunch

11:45 AM - 01:00 PM

Next Generation MSC Technologies (Industry Luncheon Hosted By RoosterBio Inc.)

Chair: Jon A. Rowley, PhD, RoosterBio Inc, United States

Speakers:

Radically Simplifying Scale-up and Clinical Translation of hMSCs and Extracellular Vesicles
Jon A. Rowley, PhD, RoosterBio Inc, United States

Engineered MSCs as Targeted Therapeutics
John Davies, PhD, DSc, Tissue Regeneration Therapeutics Inc., Canada
Chair: Jon A. Rowley, PhD, RoosterBio Inc, United States Speakers: Radically Simplifying Scale-up and Clinical Translation of hMSCs and Extracellular Vesicles Jon A. Rowley, PhD, RoosterBio Inc, Uni...
11:45 AM - 01:00 PM

Experimental Rigor in Phenotyping and Functional Testing by Flow Cytometry (Industry Luncheon Hosted By Beckman Coulter)

Speaker:

Michael Kapinsky, PhD, Beckman Coulter Life Sciences

Flow cytometry is a core method in characterization of primary human cells as the technology can provide a direct correlation between cellular identity, maturation, activation status, antigen specificity and an observed immune response. Variability of flow cytometry results may impede the method’s value, which can be mitigated through diligent validation, training and standardization, as demonstrated by The ONE Study.

To advance standardization in clinical research beyond The ONE Study gold standard, the concept of scientific rigor, as recently introduced in the NIH grant policies, may be applied that – among other aspects – enforces a robust, unbiased and replicable experimental design. Elimination of sources of human error and variability is recognized as being superior over mitigation strategies. Dry reagent formulation of technically optimized flow cytometry panels can support this goal very efficiently through exclusion of manual pipetting errors and built-in antibody panel accuracy, irrespective of time point, laboratory and operator. Further enhancement can be achieved through integration of dry reagent technology with automation, data management systems and robust flow cytometry instrumentation.
Speaker: Michael Kapinsky, PhD, Beckman Coulter Life Sciences Flow cytometry is a core method in characterization of primary human cells as the technology can provide a direct correlation between ce...
12:45 PM - 02:00 PM

Global Regulatory Perspectives (GRP) Workshop Session 3 - Clinical Strategies for Treatment of Pediatric Diseases

Moderator:

Dominic Wall, PhD, FFSc(RCPA), Director of Pathology Operations, Peter MacCallum Cancer Center, Australia

Presenters:

Rachel Witten, MD, Lead Medical Officer, FDA, United States

I-Ning Tang, MD, MS, Section Chief of Regulatory Science, Taiwan Center for Drug Evaluation, Taiwan

Jaap Jan Boelens, MD, PhD, Pediatric Hematologic Oncologist, Chief of Stem Cell Transplantation and Cell Therapy Service, United States
Moderator: Dominic Wall, PhD, FFSc(RCPA), Director of Pathology Operations, Peter MacCallum Cancer Center, Australia Presenters: Rachel Witten, MD, Lead Medical Officer, FDA, United States I-Ning ...
01:00 PM - 02:30 PM

Cell Processing Track Session 3 - Product Testing and Characterization by Flow Cytometry - A Joint Session with the International Society for Advancement of Cytometry

Co-Chairs:

Ruud Hulspas, PhD, Cellular Technologies Bioconsulting, Netherlands
Paul Wallace, PhD, Roswell Park Comprehensive Cancer Center, United States

Speakers:

Rigor & Reproducibility in Flow Cytometry
Ruud Hulspas, PhD, Cellular Technologies Bioconsulting, Netherlands

Pre and Post Product Validation of Manipulated Products
Paul Wallace, PhD, Roswell Park Comprehensive Cancer Center, United States

Phenotypic Analysis of CARs for Solid Tumours
Paul Neeson, PhD, Peter MacCallum Cancer Centre, Australia
Co-Chairs: Ruud Hulspas, PhD, Cellular Technologies Bioconsulting, Netherlands Paul Wallace, PhD, Roswell Park Comprehensive Cancer Center, United States Speakers: Rigor & Reproducibility in Flow C...
01:00 PM - 02:30 PM

Global Regulatory Perspectives (GRP) Workshop Session 3 - Treatment Impact on Pediatric Patient’s Innate Biology

Speakers
  • Jaap Jan Boelens MD, PhD, Chief; Stem Cell Transplantation and Cellular Therapies Pediatrics, Memorial Sloan Kettering Cancer Center (Speaker)
  • Ining Tang MD|MSc, Section chief, Regulatory science, Center for Drug Evaluation (Speaker)
  • Dominic Wall, CSO- Cell Therapies, Peter MacCallum Cancer Center (Moderator)
01:00 PM - 02:30 PM

ISCT-CBA Cord Blood Series in Partnership with ASBMT Session 3 - Cord Blood Immunotherapy

Chair:
Elizabeth J. Shpall, MD, MD Anderson Cancer Center, United States

Speakers:

Application of cord blood as a donor source for virus specific T cells
Catherine Bollard, MD, FRACP, FRCPA, Children's National Medical Center and George Washington University, United States 

Engineered cord blood derived NK cells for the treatment of cancer
Katy Rezvani, MD, PhD, MD Anderson Cancer Center, United States

Conditioning umbilical cord MSCs for inflammatory diseases
Yufang Shi, PhD, Institutes for Translational Medicine, China
Chair: Elizabeth J. Shpall, MD, MD Anderson Cancer Center, United States Speakers: Application of cord blood as a donor source for virus specific T cells Catherine Bollard, MD, FRACP, FRCPA, Childre...
01:00 PM - 02:30 PM

MSC Workshop Session 3 - MSC Regulatory Landscape

Session Chair: Sowmya Viswanathan, PhD, University Health Network, Canada

Speakers:

The Regulatory Landscape Around Cell Therapy and Regenerative Medicine in Japan
Yoji Sato, PhD, Division of Cell-Based Therapeutic Products, National Institute of Health, Japan

A Translational Perspective on MSC Therapies
Willem Fibbe, MD, PhD, Leiden University Medical Center, Netherlands

Regulatory landscape for cell and gene therapies in Australia
Tony Manderson, PhD, Head, Cell & Tissue Therapies Unit, TGA, Australia
Session Chair: Sowmya Viswanathan, PhD, University Health Network, Canada Speakers: The Regulatory Landscape Around Cell Therapy and Regenerative Medicine in Japan Yoji Sato, PhD, Division of Cell-...
01:00 PM - 02:30 PM

ISCT-FACT Quality Bootcamp Session 3 - Process Validation 101

Confirmed Speakers: 

Annette Trickett, PhD, Processing Facility Director, Blood and Marrow Transplant Program, Kids Cancer Centre, Sydney Children's Hospital, Australia
Robyn Rodwell, PhD, MSc (GMP & QM), Director/Scientific Director, Queensland Cord Blood Bank at the Mater, Australia
Confirmed Speakers:  Annette Trickett, PhD, Processing Facility Director, Blood and Marrow Transplant Program, Kids Cancer Centre, Sydney Children's Hospital, Australia Robyn Rodwell, PhD, MSc (GMP &...
Speakers
  • Robyn Rodwell PhD|MSc|(AIMS), FFSc(RCPA), Director/Scientific Director, Queensland Cord Blood Bank At The Mater, Mater Misericordiae Hospitals (Speaker)
  • Annette Trickett PhD, Principal Scientist, NSW Health Pathology, Randwick Hospitals (Speaker)
01:00 PM - 02:30 PM

ISCT Chief Scientific Officer Emerging Technologies Showcase

Brought to you by Dr. Daniel J Weiss, ISCT Chief Scientific Officer, and new for ISCT 2019, the Emerging Technologies Showcase aims to highlight up and coming areas in the field of cell and gene therapy clinical tranlsation. Topics to be addressed include Ophthalmology and Oral & Craniofacial Regeneration.

Chair: Daniel J. Weiss, MD, PhD, University of Vermont, United States

Speakers:

Dental stem cells in orofacial tissue regeneration
Songtao Shi, DDS, PhD, South China Center of Craniofacial Stem Cell Research, Guanghua School of Stomatoogy, Sun Yat-Sen University, China

Cellular therapies for peridontal tissue regeneration
Stan Gronthos, PhD, Adelaide Medical School, Faculty of Health and Medical Sciences, The University of Adelaide, Australia

Photoreceptor transplantation for retinal degenerative diseases
Masayo Takahashi, MD, PhD, Laboratory for Retinal Regeneration, RIKEN Center for Biosystems Dynamics Research, Japan
Brought to you by Dr. Daniel J Weiss, ISCT Chief Scientific Officer, and new for ISCT 2019, the Emerging Technologies Showcase aims to highlight up and coming areas in the field of cell and gene thera...
02:00 PM - 02:30 PM

Global Regulatory Perspectives Case Study 3

Panel Discussion:

Dominic Wall, Rachel Witten, I-Ning Tang, Jaap Jan Boelens
Panel Discussion: Dominic Wall, Rachel Witten, I-Ning Tang, Jaap Jan Boelens
02:30 PM - 02:45 PM

02:45 PM - 04:15 PM

Cell Processing Track Session 4 - Post-Infusion to Commercialization

Co-Chairs:

Mara Neal, SH(ASCP)MT, MS, MBA, Cook Regentec, USA
Vicki Antonenas, BSc, MSc, Westmead Hospital, Australia

Speakers:

Ending the Process - Clinical Challenges of Cellular Therapy Product Infusion
Kenneth Micklethwaite, MBBS, PhD, FRACP, FRCPA, Westmead Hospital, Australia

Decision Criteria and Tracking of Infused Cell Products
Virginia Litwin, PhD, Caprion Biosciences Inc., Canada

Lessons Learned in Transferring Cellular Therapy Products from Academia to Commercialization
Bruce Levine, PhD, University of Pennsylvania, United States
Co-Chairs: Mara Neal, SH(ASCP)MT, MS, MBA, Cook Regentec, USA Vicki Antonenas, BSc, MSc, Westmead Hospital, Australia Speakers: Ending the Process - Clinical Challenges of Cellular Therapy Product ...
02:45 PM - 04:15 PM

Global Regulatory Perspectives (GRP) Workshop Session 4 - Regulatory Convergence: Where we are and what we are up to?

Moderator: Karen Nichols, Esq., VP Regulatory and Quality, Magenta Therapeutics, United States

Speakers:

Judith Arcidiacono, MS, International Regulatory Expert, U.S. Food and Drug Administration, Center for Biologics Evaluation and Research, Office of Tissues and Advanced Therapies, United States

Srinvasan Kellathur, PhD Director, Advanced Therapy Products Branch, Health Sciences Authority, Singapore

Francesco Cicirello, PharmD (GPhC, FOPH), MSc, PIC/S Deputy Chair of the SCH and Chair of the Annex 2 WG, Subject Matter Expert Inspector, Therapeutic Goods Administration, Australia
Moderator: Karen Nichols, Esq., VP Regulatory and Quality, Magenta Therapeutics, United States Speakers: Judith Arcidiacono, MS, International Regulatory Expert, U.S. Food and Drug Administration, ...
02:45 PM - 04:15 PM

ISCT-CBA Cord Blood Series in Partnership with ASBMT Session 4 - Cord Blood Derived MSC, Exosome, and Regenerative Medicine

Chair:
Elizabeth J. Shpall, MD, MD Anderson Cancer Center, United States

Speakers:

Cord blood derived MSCs and exosomes for clincial use
Elizabeth J. Shpall, MD, MD Anderson Cancer Center, United States

Mesenchymal stromal cells in biological and clinical perspective
Willem Fibbe, MD, PhD, Leiden University Medical Center, Netherlands

Fact Standards for Immune Effectors and Stem Cell-Based Gene Therapy
Helen Heslop, MD, Baylor College of Medicine, United States
Chair: Elizabeth J. Shpall, MD, MD Anderson Cancer Center, United States Speakers: Cord blood derived MSCs and exosomes for clincial use Elizabeth J. Shpall, MD, MD Anderson Cancer Center, United St...
02:45 PM - 04:15 PM

MSC Pre-Conference Workshop Session 4 - Characterization and Study Design

Session Chair: Donald Phinney, PhD, Scripps Research Institute, USA

Speakers:

Epigenetic Regulation in Mesenchymal Stem Cell Immune Therapies
Songtao Shi, DDS, PhD, South China Center of Craniofacial Stem Cell Research, Guanghua School of Stomatology, Sun Yat-sen University, China

Inflammation and MSC Therapy Design
Yufang Shi, PhD, Institutes for Translational Medicine, China

The Characterization and Rational of the use of MSC on the Treatment of Fibrotic Lung Diseases: Improving Pulmonary Hypertension and the Removal of Collagen
Luis Ortiz, PhD, University of Pittsburgh, USA
Session Chair: Donald Phinney, PhD, Scripps Research Institute, USA Speakers: Epigenetic Regulation in Mesenchymal Stem Cell Immune Therapies Songtao Shi, DDS, PhD, South China Center of Craniofacia...
02:45 PM - 04:15 PM

ISCT-FACT Quality Bootcamp Session 4 - Evaluating and Reporting Audit Results

Confirmed Speakers: 

Heather J. Conway, CQA(ASQ), Quality Manager, Foundation for the Accreditation of Cellular Therapy, United States
Confirmed Speakers:  Heather J. Conway, CQA(ASQ), Quality Manager, Foundation for the Accreditation of Cellular Therapy, United States
Speakers
  • Heather Conway CQA(ASQ), Quality Manager, Foundation for the Accreditation of Cellular Therapy (Speaker)
02:45 PM - 04:15 PM

Doing Business in Korea

New for ISCT 2019, immerse yourself in Korea’s CGT industry network and learn from leaders at the forefront of this emerging cell & gene therapy market.

14:45 – 14:55 – Welcome from CARM and ISCT

So Ra Park, MD, PhD Secretary General, Council for Advanced Regenerative Medicine (CARM); Director, Strategic Center for Regenerative Medicine (SCRM); Dean/Professor, Inha University School of Medicine, Korea

John Rasko, AO, BSc(Med), MBBS(Hons), PhD, MAICD, FFSc(RCPA), FRCPA, FRACP, FAHMS, ISCT President 2018-2020; Royal Prince Alfred Hospital; University of Sydney; Centenary Institute, Australia

14:55 – 15:15 – Presentation by MoHW

In-Taek Lim, Director General, Bureau of Health Industry, MoHW, Korea

15:15 – 15:35 – Presentation by CARM

So Ra Park, MD, PhD Secretary General, Council for Advanced Regenerative Medicine (CARM); Director, Strategic Center for Regenerative Medicine (SCRM); Dean/Professor, Inha University School of Medicine, Korea

15:35 – 16:15 – Korean Company Presentations

Speakers:

Sung hyun Choi, Project Leader (PL), Daewoong Co., Ltd
Nam Ho Kim, Researcher, Biosolution co., Ltd.
Donghoon Oh, Chief Scientific Officer, STEMLAB, Inc.
San U. Song, Founder and Chief Scientific Officer, SCM Lifescience Co. Ltd
Jooyoun Lee, President of R&D Center, Xcell Therapeutics
Jong Seong Ahn, Research Center Director, Green Cross Cell corp.
Gunsoo Kim, Chief Executive Officer, Curocell Inc.
New for ISCT 2019, immerse yourself in Korea’s CGT industry network and learn from leaders at the forefront of this emerging cell & gene therapy market. 14:45 – 14:55 – Welcome from CARM and ISCT So...
Speakers
  • Jong Seong Ahn, Director of Research, Green Cross Cell Corp. (Speaker)
  • Sung Hyun Choi PhD, Project Leader, Daewoong Pharmaceuticals (Speaker)
  • GUNSOO KIM, CEO, Curocell Inc. (Speaker)
  • See More..
04:30 PM - 06:30 PM

Corporate Symposium Hosted By Miltenyi Biotec

Listen to three exciting talks giving insights into clinical applications and development of cell therapies.
• Development of automated protocols for the production of iPSC derived therapies (Amit Chandra)
• Automated manufacturing of chimeric antigen receptor T cells (Lin Yang)
• Point of care manufacturing of anti-CD19 CAR T cells for lymphoma (Marcos de Lima)
Listen to three exciting talks giving insights into clinical applications and development of cell therapies. • Development of automated protocols for the production of iPSC derived therapies (Amit Cha...
07:00 PM - 09:30 PM

ISCT 2019 President's Welcome Address and Exhibit Open House Reception

President's Welcome Address 19:00-19:30 in the Plenary Hall

Exhibit Open House Reception 19:30-21:30 in the Exhibit Hall
President's Welcome Address 19:00-19:30 in the Plenary Hall Exhibit Open House Reception 19:30-21:30 in the Exhibit Hall
07:45 PM - 08:00 PM

Establishing the CCRM Model in Australia and Beyond: Opportunities and Challenges (Corporate Product Theatre Opening Hosted By CCRM)

Speaker:
Michael May, PhD,President & CEO, Centre for Commercialization of Regenerative Medicine, Canada

CCRM is a Canadian-based commercialization and translation centre accelerating the development of revolutionary new therapies in the field of regenerative medicine and cell/gene therapy. CCRM is working closely with Australian stakeholders to replicate the CCRM model in Australia, as part of a global network of linked commercialization hubs. Dr. May’s presentation will highlight the key features of the CCRM model - key stakeholder networks, specialized teams and dedicated infrastructure - as well as showcase the major outcomes and lessons learned after eight years of operation. Finally, he will describe how global collaboration and scaling of the model is needed to achieve sustainability and generate health and economic benefits for all parties.
Speaker: Michael May, PhD,President & CEO, Centre for Commercialization of Regenerative Medicine, Canada CCRM is a Canadian-based commercialization and translation centre accelerating the development...
Thursday, May 30
07:30 AM - 08:30 AM

Hot Topic Session 1 - Bioengineering of the GI Tract

Chair: Rachele Ciccocioppo, MD, University of Verona School of Medicine, Italy

Speakers:
Advanced Stem Cell Enteric Neuropathy Therapy: Tissue-Engineering the Second Brain
Tracy Grikscheit, MD, Children's Hospital of Los Angeles, United States

The enteric nervous system (ENS) includes wide-ranging cell types in a diverse interconnected network with intimate connectivity and approximately as many neurons as the spinal cord. For this reason it is often termed the second brain. Cells in the ENS arise from vagally specified enteric neural crest cells, which can also be differentiated from human pluripotent stem cells (hPSC). Progress toward meeting the regulatory conditions for hPSC as cell therapies has been advanced by the generation of clinically acceptable good manufacturing practice compliant lines, which include appropriate donor consenting, extensive documentation of clinically acceptable reagents, and further safety testing. initial cell therapy in development in our lab of Advanced Stem Cell Enteric Neuropathy Therapy, or ASCENT, might be of use in the future human therapy of enteric neuropathies.

Regenerative Medicine Technologies Technologies Applied to Beta Cell Replacement
Giuseppe Orlando, MD, PhD, Wake Forest University Baptist Medical Center, USA

Learning Objectives:
• To illustrate the state of the art of beta cell replacement therapies for the treatment of type 1 diabetes
• To explain how regenerative medicine technologies may impact the field of beta cell replacement
Chair: Rachele Ciccocioppo, MD, University of Verona School of Medicine, Italy Speakers: Advanced Stem Cell Enteric Neuropathy Therapy: Tissue-Engineering the Second Brain Tracy Grikscheit, MD, Ch...
07:30 AM - 08:30 AM

Hot Topic Session 2 - Getting to the Truth – Designing Studies and Clinical Networks for Objective Evaluation of Cellular Therapies for Osteoarthritis of the Knee

This Hot Topics Session is hosted by the newly formed Committee on Orthopaedics and Musculoskeletal Cell Therapies within the International Society for Cell & Gene Therapy (ISCT).

Our mission is to advance the field by serving as a catalyzing forum, particularly in the areas of:

1) Development of collaborative international networks of clinicians and scientists working in Musculoskeletal Cell Therapies.
2) Facilitating the design and execution of rigorous pre-clinical studies and clinical studies and registries.
3) Development and use of objective standards in data collection and communication of the outcomes of clinical trials.
4) Development and validation of safe and effective methods of clinical cell manufacturing.
5) Development of inter-disciplinary communication and standards in nomenclature related to cellular therapies.
6) Develop programs for the ISCT Annual meeting and regional meetings organized by regional ISCT Chapters in North America, Europe and Asia.

In this Hot Topics Session, leaders from Europe, Japan and North America will provide an overview of the current status of translation of cellular therapies into prospective musculoskeletal registries and clinical trials in these distinct regions of the world. We will particularly explore the unique differences in clinical target opportunities, funding strategies, and regulatory pathways between these regions of the world, and seek to identify opportunities for collaborative synergy and best practice modeling.

Chair:
George Muschler, MD , Cleveland Clinic, United States

Speakers:
Nakamura Norimasa, MD, PhD, Osaka Health Science University, Japan

Designing Optimal Studies of Cellular Therapies in the Knee
Danièle Noël, PhD, Inserm Institute for Regenerative Medicine and Biotherapies, France

Constance Chu, MD, Stanford University School of Medicine, USA
This Hot Topics Session is hosted by the newly formed Committee on Orthopaedics and Musculoskeletal Cell Therapies within the International Society for Cell & Gene Therapy (ISCT). Our mission is to a...
07:30 AM - 08:30 AM

Commercial, Manufacturing and Clinical Aspects of a Multinational Rare Disease Gene Therapy Trial (Corporate Breakfast Tutorial Hosted By Cell Therapies Pty Ltd)

Chair: Nathan Smith Project Portfolio Manager, Cell Therapies Pty Ltd, Australia

Speakers:

Geoff Mackay Chief Executive Officer, AvroBio, United States

Alexandra Rizzitelli, PhD Cell Therapy Specialist, Cell Therapies Pty Ltd, Australia

David Ritchie, MD, PhD Haematologist & Head of Allogeneic Stem Cell Transplantation, The Royal Melbourne Hospital, Australia

Conducting a multinational rare disease gene therapy clinical trial poses many challenges that must be overcome to ensure a successful clinical study. We will present a case study detailing the different aspects to the implementation of AVROBIO’s phase 1/2 clinical study for their AVR-RD-01 product for patients with Fabry disease in Australia.
Initially, AVROBIO, a leader in lentiviral-based gene therapies, will review their corporate goals, the technology platform, the product, and the disease. Cell Therapies, a contract manufacturer based in Melbourne that supplies the clinical product, will provide details on the technology transfer and product manufacturing. Finally, the clinical aspects and patient considerations of the Fabry disease
study will be outlined by The Royal Melbourne Hospital, who has been treating patients in Victoria for over 170 years.
Chair: Nathan Smith Project Portfolio Manager, Cell Therapies Pty Ltd, Australia Speakers: Geoff Mackay Chief Executive Officer, AvroBio, United States Alexandra Rizzitelli, PhD Cell Therapy Spe...
07:30 AM - 08:30 AM

Overcoming Challenges in Cell Therapy Manufacturing (Corporate Tutorial hosted by Thermo Fisher Scientific)

Chair: Randy Schweickart, Director of Engineering, Juno Therapeutics, Inc

Speaker: Tia Hexom, Field Applications Scientist, Thermo Fisher Scientific

There are multiple challenges in the manufacture of T cell therapies that must be solved in order to meet the global commercialization need for these therapies. In this presentation, solutions to some of these challenges will be presented, including how to reduce variability in the donor’s starting material by simultaneous isolation and activation of naïve and early memory T cells, which persists in vivo. Also, a solution to improve productivity and scalability with be presented that is xeno-free, serum- free, and scalable to meet workflow processes. Lastly, an industry leader, Juno Therapeutics, will discuss some key process challenges and mitigations to ensure robust and efficient commercial cell therapy manufacturing.
Chair: Randy Schweickart, Director of Engineering, Juno Therapeutics, Inc Speaker: Tia Hexom, Field Applications Scientist, Thermo Fisher Scientific There are multiple challenges in the manufacture ...
09:00 AM - 10:30 AM

Presidential Plenary - CAR-Ts Beyond B Cells

CAR-T cell therapies for B-cell malignancies are now approved by regulators and governments in many advanced markets worldwide. They are changing lives and providing cures for those suffering with specific types of relapsed or refractory leukaemias and lymphomas. While CAR-T cells against B-cell markers are proven and will likely be used more widely for additional blood cell malignancies, progress to date concerning solid tumours has been less spectacular. In this session three outstanding clinician-scientists from three continents will discuss their various approaches to using cellular immune-gene therapies for non-B cell cancers. In the context of using CAR-T cells “beyond B Cells”, Dr Gill will discuss his approach to myeloid malignancies, Dr Han will provide data from his Phase 1 trial in mesothelin-positive cancers, and Dr Locatelli will outline his innovative strategy for neuroblastoma. The challenges required to overcome a less-conducive cellular microenvironment will also be defined.

CAR T Cells for Acute Myeloid Leukemia
Saar Gill MD, PhD, University of Pennsylvania, USA
In this presentation, I will review the reasons CAR T cell therapy for myeloid malignancies has lagged behind that for B-lymphoid malignancies and outline several potential ways to bring this potent therapeutic modality to patients.

Treatment of Autologous Mesothelin-targeted CAR T Cells with Genomic PD-1/TCR Deletion in Solid Tumor Patients
Weidong Han MD, PhD , Chinese PLA General Hospital, China
Experimental data both at cellular and tumor-bearing mice levels revealed an enhanced anti-tumor effect of mesothelin-redirected CAR T cells after deleting genomic PD-1, meanwhile this effect and the CAR T cell expansion capacity is not disrupted by TCR knock out. Then, dose-escalated mesothelin-PD1/TCR-KO-CAR T cell infusion (1× to 90×105/kg) regimen was translated into phase I clinical trial in advanced mesothelin+ solid tumor patients beginning from Feb. 2018 (NCT03545815). Infusion-related common adverse events including febrile syndrome, anaphylaxis, CRS, newly-presented cavity effusion were not observed, and the suspicious indications of autoimmune reaction were not perceived in all 16 patients. No aberrant implications unrelated to tumor progression were perceived as well. Six patients had experienced accrued effusions in tumor-infiltrated cavity within 3 weeks after cell infusions. CAR T cell monitoring showed a CAR-DNA copy number peak within 2 weeks and undetectable 4 weeks after infusions in PB; interestingly, TCR+, but not TCR- CAR+ T cells in effusions; sparsely detectable only in 2 tumor biopsies. DNA copy number comparison did not indicate a superior existence of CAR T cells in tumors than in PB. Combined data with clinical response implied an inferior anti-tumor activity of CAR T cells with PD-1 and/or TCR deletion.

CAR-T cells in Solid Tumors: The Model of Neuroblastoma
Franco Locatelli MD, PhD , University of Pavia and IRCCS Ospedale Pediatrico Bambino Gesù, Italy
Autologous T cells transduced with CD19-directed chimeric antigen receptors (CAR) have recently been approved by a several regulatory agencies for the treatment of relapsed/refractory leukemia and lymphoma in view of their outstanding efficacy. The efficacy of the approach in solid tumors is much less consolidated, mainly because of greater challenges largely represented by the immune-suppressive effects displayed by the tumor microenvironment and by the difficulty that
CAR -T cells have to penetrate a solid mass. Neuroblastoma is the most extracranial common solid tumor of childhood with a still unsatisfactory prognosis for patients with metastatic disease or with genetic lesions predicting a high risk of recurrence. Results of the phase I/II academic clinical trial on GD2-specific, third-generation CAR-T cells in children with relapsed/refractory neuroblastoma will be presented, using this model for discussing the hurdles to be overcome for successfully translating the CAR-T cells approach in solid tumors.
CAR-T cell therapies for B-cell malignancies are now approved by regulators and governments in many advanced markets worldwide. They are changing lives and providing cures for those suffering with spe...
10:30 AM - 10:45 AM

Solutions That Enable Efficiencies in cGMP Manufacturing (Global Showcase Presentation) Hosted By GE Healthcare

Speaker:
Greg Crescenzi ,Enterprise Solutions Leader, GEHC Cell and Gene Therapy, United States

GE Healthcare’s cell and gene therapy flexible and scalable manufacturing solution with the FlexFactory™ platform is intended to improve cell therapy manufacturing. Working with customers GE provides drug developers with a flexible and scalable manufacturing platform suitable for early-stage clinical or commercial production. This session will provide an overview of some of the key manufacturing challenges associated with cell therapy and discuss how this new solution can help mitigate some of these challenges and reduce time to market.
Speaker: Greg Crescenzi ,Enterprise Solutions Leader, GEHC Cell and Gene Therapy, United States GE Healthcare’s cell and gene therapy flexible and scalable manufacturing solution with the FlexFactory...
10:30 AM - 11:00 AM

Coffee Break with Exhibits

10:45 AM - 11:00 AM

Engineered for Life – BIOSTAT ® RM TX with FLEXSAFE® RM TX For Culturing Consistent Quality Cellular Products (Global Showcase Presentation Hosted By Sartorius Stedim Biotech)

Speaker:
Ruth McDermott, Platform Manager Regenerative Medicine, Sartorius Stedim Biotech, United Kingdom

Sartorius has recently launched its new single used rocking motion bioreactor platform for the manufacture of cell therapy products.

The BIOSTAT® RM TX bioreactor system combined with Flexsafe® technology enables the ex vivo expansion of patient cells for cell immunotherapy applications. We will present how this platform has been designed for:
• Consistent cell growth
• Minimized risk of contamination
• Improved manufacturing efficiency
• Innovative cell harvesting
Speaker: Ruth McDermott, Platform Manager Regenerative Medicine, Sartorius Stedim Biotech, United Kingdom Sartorius has recently launched its new single used rocking motion bioreactor platform for th...
11:00 AM - 12:15 PM

Plenary 1 Satellite Session - Immunotherapy Oral Abstract Presentations I

Chair: Saar Gill, MD, PhD, FRACP Assistant Professor, University of Pennsylvania, USA

Selected Oral Abstract Presentations

David Bishop (AU) – Matched Sibling Donor-Derived Piggybac Car19 T Cells Induce Remission of Relapsed/Refractory Cd19+ Malignancy Following Haematopoietic Stem Cell Transplant (Abstract 1)

Cynthia Bamdad (US) – First-In-Human CAR T for Solid Tumors Targets the MUC1 Transmembrane Cleavage Product (Abstract 2)

Patrick Hanley (US) – Hexaviral Specific T-Cells Targeting Parainfluenza, CMV, EBV, Adenovirus, HHV6 and BKV Used for Prophylaxis and Treatment of Viral Infections in Patients Post Stem Cell Transplant (Abstract 3)

Zwi Berneman (BE) – Vaccination of Cancer Patients with Dendritic Cells Electroporated with mRNA Encoding the Wilms' Tumor 1 Protein (WT1): Correlation of Clinical Effect and Overall Survival with T-cell Response (Abstract 4)

Zhen Zeng (CN) – Phase I Dose Escalating Trail of GPC3-Targeted CAR-T Cells by Intratumor Injection for Advanced Hepatocellular Carcinoma (Abstract 5)
Chair: Saar Gill, MD, PhD, FRACP Assistant Professor, University of Pennsylvania, USA Selected Oral Abstract Presentations David Bishop (AU) – Matched Sibling Donor-Derived Piggybac Car19 T Cells In...
11:00 AM - 12:15 PM

Plenary 2 Satellite Session – Enabling Technologies in Regenerative Medicine

Chair: Tracy Grikscheit (US)

Cell Fiber Technology for 3D Tissue Construction and Cell Therapy
Shoji Takeuchi (JP)

Learning objectives, to understand:
• The cell fiber technology and its advantages
• How to form the cell fiber
• The application of the cell fiber in 3D cell culture and cell therapy

Engineering the Extracellular Microenvironment to Modulate the Therapeutic Properties of Mesenchymal Stromal Cells
Jessica Frith (AU)

Mesenchymal stem/stromal cells (MSCs) show great promise for a wide range of applications including treating graft-vs-host disease, rheumatoid arthritis and cardiovascular diseases, as well as in the tissue-engineering of bone and cartilage. Typical strategies for MSC-based tissue regeneration require significant expansion of the cells in vitro, most commonly using standard tissue culture flasks. However, it is increasingly understood that MSCs are highly sensitive to their extracellular environment and that physical cues from the surrounding microenvironment have a huge influence on the properties of the cells. A thorough understanding of these changes and the mechanisms that drive them therefore has the potential to underpin better ways to both expand and deliver MSCs for therapeutic applications.
We have used biomaterial systems to investigate the effects of different physical cues on the properties of MSCs and understand how mechanotransductive signalling mediates these changes. Our work has shown how miRNA signalling changes in response to the stiffness of the culture substrate and demonstrated that an understanding of these changes can be harnessed to control cell fate. We have also investigated the effects of substrate stiffness on MSC paracrine signalling, showing changes that affect the activity of the MSCs themselves as well as other cell populations, such as macrophages and T-cells, that mediate some of the immunomodulatory effects of MSCs. Investigating a light-curable PEG-gelatin hydrogel for encapsulation and delivery of MSCs, we also highlight the significant differences in cell behaviour that can occur in response to culture format, showing changes in cell migration and differentiation when MSCs are encapsulated in microgels compared to bulk hydrogels. Together these studies provide insights into the role of the extracellular environment in regulating MSC properties and provide examples of how an understanding of these processes can be applied for cell expansion and delivery.


Selected Oral Abstract Presentation:

Lucy Luo (CA) – Generating Biomimetic Mineralized Collagen Scaffolds for Bone Regeneration (Abstract 11)
Chair: Tracy Grikscheit (US) Cell Fiber Technology for 3D Tissue Construction and Cell Therapy Shoji Takeuchi (JP) Learning objectives, to understand: • The cell fiber technology and its advantag...
11:00 AM - 12:15 PM

Strategies for Commercialization Track Session 1 - Think Big and Think Early to Prevent Failure at Commercial Scale

The impact of failure within platforms like manufacturing, clinical, logistics, can all prevent a patient from receiving their treatment and a developer either being reimbursed or generating clinical data. All these parts of the therapies value chain are interlinked, meaning that if you make a decision in one area it impacts all the others (e.g. developing cryopreservation enables a single manufacturing site and optimises logistics, but means you have to manage a lower cell viability, develop an in clinic thawing strategy).

To manage this, it is critical to have a vision of what the final, commercial scale, strategy is going to look like. Therapy developers can then align process, clinical, manufacturing, logistics platform strategies to build, test and optimise through clinical trials. The value of this, forward thinking, has been proven by the application of Quality by Design principles within process development. There is now, a recently published, concept of Logistics by Design that has the same forward thinking, risk based approach. All of which means that therapy developers now have the opportunity to plan the development of their entire value chain.

Using current manufacturing exemplars, this session will investigate the value of planning early. The speakers are all from a technical backgrounds and will look to provide practical advice to help therapy developers build viable platforms.

Chair:
Simon Ellison, MBA, World Courier, UK

Speakers:

Data Capture and Analysis Through the Cell Therapy Value Chain
Heidi Hagen, MS, MBA, Vineti, Inc, USA

Learning objectives:
• How can you manage and control the value chain from order to infusion for cell therapy
• The data captured throughout the value chain during clinical trials will establish the parameters of your commercial operations
• Demonstrate how complex work flows, multiple points of data capture, and intervention by multiple stakeholders in disparate geographies can be orchestrated successfully and enable your product to have a high trust level by physicians and patients.

Plans for Global Roll Out of An Off-The-Shelf Allogeneic Cellular Therapy
Kilian Kelly, PhD, Cynata Therapeutics, Australia

Learning objectives:
• Regional distribution - import/export and use of depots
• Site/patient level distribution, local storage and just-in-time delivery
• Point of care considerations

Plan to Purpose
Andrew McDonald, World Courier, Australia

Learning objectives:
• Explore the logistics stages involved for your production cycle and ensure they are commensurate with your objectives
• Investigate the possible pitfalls and engage contingencies to ensure continuity and compliance
• Create, optimise and execute the plan to deliver healthier futures

Considerations for Cell and Gene Manufacturing
Robin Ng, PhD, Cellular Biomedicine Group, China
- How operations can drive efficiency
-Managing supply chain is critical for the success of delivery therapy to the patients
-Manufacturing needs to consider both the availability and affordability to the patients
The impact of failure within platforms like manufacturing, clinical, logistics, can all prevent a patient from receiving their treatment and a developer either being reimbursed or generating clinical ...
11:00 AM - 12:15 PM

Quality and Operations Track Session 1 – Emerging Standards in Cell & Gene Therapies

Chair: Sowmya Viswanathan, PhD, University Health Network, Canada

Speakers:

Building the Foundation for Regenerative Medicine Innovation through Standards
Judith Arcidiacono, PhD, FDA, United States

Learning objectives:
• Define the types of standards that can be utilized in the development and testing of cell and gene therapies
• Describe how standards developed
• Describe how standards can be used to support regulatory applications to FDA for cell and gene therapy products

Development of a Cryopreservation Standard to Speed Cell Therapy/Regenerative Medicine Product Development
Brian J. Hawkins, PhD, Pluristyx Inc, United States

Learning objectives:
• Discuss the role of cryopreservation for cell therapy/regenerative medicine product development
• Demonstrate how cryopreservation best practices can be incorporated into a cell manufacturing workflow
• Provide an update on the current ANSI/PDA Cryopreservation Standards Initiative

FACT Accreditation Requirements
Paul Eldridge, UNC Lineberger Advanced Cellular Therapeutics Facility, Chapel Hill, United States
Chair: Sowmya Viswanathan, PhD, University Health Network, Canada Speakers: Building the Foundation for Regenerative Medicine Innovation through Standards Judith Arcidiacono, PhD, FDA, United Stat...
11:00 AM - 12:15 PM

Advanced Practice Professionals (APP track) Session 1 – Cord-Blood Derived Therapies for Regenerative Medicine, Transplantation & Cancer Immunotherapy

Co-Chairs:
Megan Cornelison, MS, PA-C, MD Anderson Cancer Center, USA
Tobi Fisher, MPAS, PA-C, MD Anderson Cancer Center, USA

This session will discuss the benefits of cord blood transplantation and ex vivo expansion in allotransplantation, and the novel use of cord-blood derived iPSCs (immunologic Pluripotent stem cells). We will also be discussing CAR T-cell and gene therapy development and manufacturing.

Speakers:
Cord Blood Transplantation for Hematologic Malignancies: Do We Need Expansion?
Juliet Barker, MBBS, Memorial Sloan Kettering Cancer Center, United States

Generating GMP-grade iPSCs from Banked Cord Blood for Potential Regenerative Therapy Purposes
Ngaire Elwood, PhD, Murdoch Children's Research Institute, Australia

CAR-T Cell and Gene Therapy Development and Manufacturing
David DiGiusto, PhD, Semma Therapeutics, United States
Co-Chairs: Megan Cornelison, MS, PA-C, MD Anderson Cancer Center, USA Tobi Fisher, MPAS, PA-C, MD Anderson Cancer Center, USA This session will discuss the benefits of cord blood transplantation an...
11:00 AM - 12:15 PM

Regulatory Spotlight - Clinical Site Cell Supply for Commercial CAR-T Products - How to Integrate Regional Rules and Policies into Global Networks

All the commercial CAR-T programs either approved/seeking approval are dependent for supply from clinical apheresis and cryopreservation services. This has raised myriad issues, not the least of which is the diversity of regulatory oversight and control in the markets where these products are already approved- EU, US, Canada and Australia. In some markets this is considered to be an essentially clinical activity with limited regulatory interaction other than meeting starting material specifications, in others this is covered by existing tissue practice standards which are variably regulated, whilst in Australia this is seen as a manufacturing step that requires the manufacturer to demonstrate they can exert control on some minimum quality and testing requirements. We propose to engage with regulators and industry to discuss their strategy in their relevant markets to foster a discussion on what a uniform requirement should look like for industry and clinical centers.

Chair: Simon Harrison, (AU)

Speakers:
Francesco Cicirello (AU)
Bryan Silvey (US)
Peter Holman (US)
All the commercial CAR-T programs either approved/seeking approval are dependent for supply from clinical apheresis and cryopreservation services. This has raised myriad issues, not the least of which...
12:15 PM - 01:45 PM

Lunch with Exhibits

12:30 PM - 12:45 PM

Beyond Blood - Supporting Cell Therapies From The Bottom Up (Global Showcase Presentation Hosted By Australian Red Cross Blood Service)

Speakers:
Shane Winzar , Australian Red Cross Blood Service, Australia
Augustin Raj , Australian Red Cross Blood Service, Australia

The Blood service has for many decades provided key support services to Australia’s Cell Therapy, Precision Medicine and Regenerative Medicine industries, from public based research through to fully scaled commercial endeavors.
Our services and capabilities include provision of research grade blood components, set up of quality systems, Therapeutic Good licensing consultancy, screening of autologous and allogeneic donors, for HLA typing using Next Gen sequencing and other world class platforms. Additionally, we assist cGMP collections for provision of cell therapy based clinical trials.
The Blood Service is involved in collaborative R&D in CAR-T clinical trials and provides services direct to other clients to assist them in bringing their products to provide a greater contribution to healthcare for all Australians.
Speakers: Shane Winzar , Australian Red Cross Blood Service, Australia Augustin Raj , Australian Red Cross Blood Service, Australia The Blood service has for many decades provided key support servic...
12:30 PM - 01:30 PM

Driving Speed to Market Through Collaboration (Corporate Tutorial Hosted By GE Healthcare)

Moderator:
Phil Vanek, PhD,GM, Growth Strategy, GEHC Cell and Gene Therapy, United States

Speakers:
Robin Ng, PhD, Strategy Lead, Technology and Manufacturing, Cellular Biomedicine Group, China

Rohin Iyer, PhD, Development Manager, Cell and Gene Therapy, GEHC/FastTrak@CCRM, Canada

Greg Crescenzi Enterprise Solutions Leader, GEHC Cell and Gene Therapy, United States

Effective process development and robust manufacturing are key to scaling cell and gene therapies for commercialization. A flexible solution that offers both could provide a competitive advantage. Recently, Cellular Biomedicine Group (CBMG), a clinical stage biopharmaceutical company in Shanghai, China, partnered with GE Healthcare (GEHC)’s Enterprise Solutions team as well as the Centre for Commercialization of Regenerative Medicine (CCRM) in Toronto to develop a next-generation CAR-T process based on GE’s FlexFactory equipment. Through a panel discussion they will discuss how these three teams worked together, with specific emphasis on how important logistical, technical and communication decisions were made to build a state-of-the-art CAR-T manufacturing facility/process that met CBMG’s specifications and supported their IND filing to the Chinese FDA.
Moderator: Phil Vanek, PhD,GM, Growth Strategy, GEHC Cell and Gene Therapy, United States Speakers: Robin Ng, PhD, Strategy Lead, Technology and Manufacturing, Cellular Biomedicine Group, China Roh...
12:45 PM - 01:00 PM

Regeneus' Novel Allogenic Stem Cell Platforms For Musculoskeletal and Neuropathic Pain Disorders (Global Showcase Presentation Hosted By Regeneus Ltd)

Speaker:
Charlotte Morgan, PhD , Head of R&D, Regeneus Ltd, Australia

Regeneus is an Australian based clinical stage regenerative medicine company, focused on musculoskeletal and Neuropathic pain indications. Our leading technology platform Progenza is an Adipose derived MSC product combined with MSC secretions. A Phase 1 clinical study in Knee Osteoarthritis met primary and secondary endpoints in pain reduction and positive signs of disease modification. Positive pre-clinical pain study data has also been achieved in preparation for a clinical study in Neuropathic pain.
Speaker: Charlotte Morgan, PhD , Head of R&D, Regeneus Ltd, Australia Regeneus is an Australian based clinical stage regenerative medicine company, focused on musculoskeletal and Neuropathic pain in...
01:00 PM - 01:15 PM

HPL Pathogen Reduction Treatment and Risk Assessment (Global Showcase Presentation Hosted By AventaCell Biomedical)

Speaker:
William Milligan, VP Business Development, AventaCell Biomedical Corp.; Chair, ISCT Business Models and Investment; Committee Member, ISCT-AABB Joint Working Group on Human Platelet Lysate, Canada

There is an increasing acceptance of replacing FBS with human platelet lysate as a serum-free supplement for growing cells. Human platelet lysate has proven to be a cost-effective cell culture alternative which provides greater yields of high quality cells when compared to FBS and serum-free mediums. Many clinical trials are being conducted using MSC, fibroblasts, dendritic cells and other cells grown in human platelet lysate. However, as the platelets are collected from allogeneic donors, there are concerns about the potential risk of transmitting pathogens. This session will review a current summary of pathogen reduction treatment (PRT) options for human platelet lysates with a focus on gamma irradiation and the resulting assessment of risk for pathogen transmission.
Speaker: William Milligan, VP Business Development, AventaCell Biomedical Corp.; Chair, ISCT Business Models and Investment; Committee Member, ISCT-AABB Joint Working Group on Human Platelet Lysate, ...
01:15 PM - 01:30 PM

Fast Forward to hMSCs Manufacturing: A Novel, Xeno-Free Medium Supporting Planar and Suspension Formats For Easy Scale-Up (Global Showcase Presentation Hosted By Merck)

Speaker:
Kathleen Ongena, PhD , Head of Biomaterials and Assays, Cell Therapy Bioprocessing, Merck, United States

The long-term outlook for stem cell therapy predicts an increased need for better defined media and high quality raw materials. Human mesenchymal stromal/stem cells (hMSCs) are an attractive target for clinical study as therapeutic agents. Large scale manufacturing of these adherent-dependent cells necessitates adoption of technologies such as stirred tank bioreactors where suspension culture using microcarriers is enabled. Cell culture media and supplements are critical factors of the scale-up process. Many processes currently include fetal bovine serum (FBS), a commonly-used supplement associated with regulatory, supply, and consistency challenges.

Here, we show the performance of a novel, xeno-free cell culture media formulation developed for the expansion of hMSCs in multiple cell culture platforms. We highlight the growth and characterization of hMSCs in planar, spinner, and stirred tank bioreactor growth platforms. Results indicate the medium delivers improved growth performance compared to a benchmark medium while maintaining expected characteristics of multipotency.

This xeno-free medium developed for hMSC expansion supports the immediate need for suspension-based clinical production as well as future implementation of large scale, commercial manufacturing solutions for allogeneic therapies that will be required
following clinical success.
Speaker: Kathleen Ongena, PhD , Head of Biomaterials and Assays, Cell Therapy Bioprocessing, Merck, United States The long-term outlook for stem cell therapy predicts an increased need for better de...
01:45 PM - 03:15 PM

Plenary Session 2 – Complex Cellular Structures: Realising The Regenerative Potential of Cells

Co-Chairs:
Oscar K. Lee MD, PhD, National Yang-Ming University, Taiwan
Janet Macpherson PhD, GE Healthcare Life Sciences, Australia

Speakers:

Advancing the Applications of Human Pluripotent Stem Cell-Derived Kidney Organoids
Melissa Little, PhD, Murdoch Children's Research Institute, Australia

The development of protocols for the differentiation of human pluripotent cells to complex multicellular organoids provides novel opportunities for stem cell medicine. We have developed a protocol for the generation of multicellular human kidney organoids from human pluripotent stem cells (Takasato et al, Nature, 2015; Nature Protocols, 2016). The application of kidney organoids for disease modelling, drug screening or tissue therapy options will require evidence that kidney organoids are an accurate model of the developing human kidney tissue and that the protocols for generation of such tissue are robust, transferable, able to be scaled and can result in a functional tissue. Using CRISPR/Cas9 gene edited iPSC lines, we are comprehensively examining both the lineage relationships of the cell types within kidney organoids and the identity of the component cells and comparing these to what is known in both developing mouse and human fetal tissues. Via single cell transcriptional profiling, we have examined the cellular complexity of kidney organoids, identifying the underlying sources of batch variation and the identity of potential off target cell types. Using changes in culture format, we are developing approaches for the scale up of tissue generation. Transplantation of these human pluripotent stem cell-derived kidney organoids has revealed evidence for vascularisation and maturation, as evidenced by improved ultrastructure. Altogether, these advances are moving us closer to the application of patient-derived kidney organoids for tissue regeneration.

Tissue-Engineering the Gastrointestinal Tract from Adult and Human Pluripotent Stem Cells
Tracy Grikscheit, MD, Children's Hospital of Los Angeles, USA

The availability of novel stem cell therapies is one of the most exciting, imminent changes in medicine and surgery this decade. Currently, surgical resection or correction of a congenital malformation often requires either tissue substitution or implantation of a foreign body, and both have serious disadvantages. In the case of small children, there may not be adequate tissue available for substitution. For biomaterials, three problems headline the list of deficits: mechanical or material failure, inadequate size as the child grows, and the lack of true biologic replacement. Organ transplantation has demonstrated the utility of physiologic replacement of form and function, but is still restricted by supply, morbidities of immunosuppression, and outcomes. The promise of stem/progenitor cell therapy is to improve in any or many of these categories, for example by off-the-shelf availability, reduction or removal of immunosuppression, improved longevity, and enhanced or more predictable biology. Tissue-engineering the gastrointestinal tract and its components is the next frontier for adult and pluripotent stem cell therapies.

Clinical Application of Engineering Cell Sheet for Cardiac Repair
Yoshiki Sawa, MD, PhD, Osaka University, Japan

Although LVAD implantation and Heart transplantation have been well accepted as the ultimate lifesaving means of supporting end-staged heart failure patients, there are some limitations in such therapies. So we developed cell sheet technology experimentally and introduced this to the treatment of severely damaged myocardium as translational research.
In a series of pre-clinical experiments, we proved that myoblast sheets could heal the impaired heart mainly by cytokine paracrine effect in cardiomyopathy model. We applied myoblast sheets to 4 DCM patient receiving LVAD and 2 patients showed the recovery from LVAD. Although we implanted myoblast sheet to 17 heart failure patients with ischemic etiology and majority in patients showed improvement of systolic function with ameliorated exercise tolerance and symptoms. Although in 24 NIDCM patients who were received myoblast sheet functional recovery was limited, good clinical outcome in selected NIDCM patients with preoperatively preserved diastolic function and ability of protein synthesis in myocytes accessed by expression of histone H3 lysine 4 trimethylation. In biopsy sample. Recently myoblast sheet was approved by the government as “Heart Sheet” in the treatment for ischemic cardiomyopathy.
To supply cardiomyocytes to the distressed myocardium we have developed human iPS cell derived cardiomyocyte sheet and obtained Proof of Concept with evidence of synchronous movement with recipient myocardium. And also we have established large culture system and checked safety of GMP grade iPS cell derived cardiomyocyte sheets for clinical trial by the development in new method for removal of immature iPS cells.
Regenerative technology has some potentials in the clinical treatment of heart failure which has little response to the internal medical or conventional surgical treatment and these technologies may open new era in the treatment of severely damaged myocardium.
Co-Chairs: Oscar K. Lee MD, PhD, National Yang-Ming University, Taiwan Janet Macpherson PhD, GE Healthcare Life Sciences, Australia Speakers: Advancing the Applications of Human Pluripotent Stem Cel...
03:15 PM - 03:30 PM

Industrializing Cell and Gene Therapy: Upstream Technologies (Global Showcase Presentation Hosted By Pall)

Speaker:
Seunghoon Choe, BSc., Meng. , Bioprocess Applications Specialist, Pall Biotech, South Korea

As cell and gene therapy develops more and more, there is a great need to scale up the process to produce as many cells and viruses as possible. Current 2D flatware (Roller, Cell Factories etc.) have limitation for this: huge footprint, many aseptic manipulations, many operators, and no real automation nor control. Therefore, alternatives must be found, keeping cost control and ensuring a fast scale up to hit the market quickly.

Pall has multiple technology solutions for upstream cell manufacturing, going over single-use Stirred tank bioreactors that support virus production from suspension cell culture, and adherent cell culture solutions, including multistacked bioreactors, fixed-bed bioreactors, and microcarriers. The focus for this presentation is to give a general introduction of these bioreactors from PALL and how these can be the solutions to your manufacturing and production constraints.
Speaker: Seunghoon Choe, BSc., Meng. , Bioprocess Applications Specialist, Pall Biotech, South Korea As cell and gene therapy develops more and more, there is a great need to scale up the process to ...
03:15 PM - 03:45 PM

Coffee Break with Exhibits

03:30 PM - 03:45 PM

A novel, single-use bioreactor system for expansion of human mesenchymal stem/stromal cell (Global Showcase Presentation hosted by Pall Biotech)

Speaker:
Kaitlynn Bayne, Associate Scientist, Pall Biotech, United States

There is a significant need for efficient systems that can be used to generate primary cells and stem cells that can be readily implemented in research laboratories to expedite process development studies and clinical testing. We have previously demonstrated efficient expansion of human mesenchymal stem/stromal cells (hMSC) in the PadReactor® single-use bioreactor system to the 40 L scale. Here we extend these finding by employing these components in a novel bioreactor system. The Allegro™ STR bioreactor is a new stirred tank, single-use bioreactor platform which is scalable, compact, ergonomic and designed to maximize usability and process assurance. Excellent results were achieved propagating hMSC on microcarriers in the Allegro STR single-use bioreactor over a six day culture period. Cells harvested from microcarriers at the end of the culture reached a cell concentration of 0.79 B cells/L and were 97% viable. A total of 22 B cells were harvested. Importantly, the cells retained critical quality attributes after harvest when examined in standard cell characterization assays. Results achieved in this study were also comparable to those obtained previously in the PadReactor platform. In this study we demonstrated utilization of a novel, single-use platform for efficient generation of high quality cells for process development studies and clinical testing.
Speaker: Kaitlynn Bayne, Associate Scientist, Pall Biotech, United States There is a significant need for efficient systems that can be used to generate primary cells and stem cells that can be readi...
03:45 PM - 05:00 PM

Plenary 1 Satellite Session – Immunotherapy Oral Abstract Presentations II

Selected Oral Abstract Presentations

Sophia Schreiber (DE)Characterization of MHC Class II-Restricted T-Cell Receptors For T-Cell Therapy of HBV Infection (Abstract 6)

Fenlu Zhu (US)Results of Point-Of-Care Manufacturing of Bispecific Chimeric Antigen Receptor (CAR) Lv20.19 CAR-T Cells in A Phase I Study for Relapsed/Refractory (R/R), Non-Hodgkin Lymphoma (NHL) (Abstract 7)

Blake Aftab (US)CD19 Chimeric Antigen Receptor (CAR) Engineered Epstein-Barr Virus (EBV) Specific T cells – An Off-the-Shelf, Allogeneic CAR T-Cell Immunotherapy Platform (Abstract 8)

Avery Posey (US)Generation of CAR T Cells Targeting Oncogenic TROP2 for the Elimination of Epithelial Malignancies (Abstract 9)

Belinda Kramer (AU)Targeting Tumor Associated Antigens Expressed in Osteosarcoma and Ewing's Sarcoma With CAR-T Cells (Abstract 10)
Selected Oral Abstract Presentations Sophia Schreiber (DE) – Characterization of MHC Class II-Restricted T-Cell Receptors For T-Cell Therapy of HBV Infection (Abstract 6) Fenlu Zhu (US) – Results o...
03:45 PM - 05:00 PM

Plenary 2 Satellite Session – Organoids for Regenerative Medicine

Speakers:

Generating Human T cells in Artificial Thymic Organoids
Gay Crooks, MBBS, University of California, Los Angeles, USA

Learning objectives:
• Learn how T cells develop from hematopoietic stem cells in a normal thymus
• Understand the key differences between the monolayer and 3D artificial thymic organoid (ATO) models
• Learn how tumor antigen-specific T cells can be generated in ATOs from hematopoietic and pluripotent stem cells

Pluripotent Stem Cell Models of Heart Development and Disease
David Elliott, PhD, Murdoch Children's Research Institute, Australia

Learning objectives:
• Human pluripotent stem derived cardiomyocytes are important tools to understand disease.
• Isogenic cell lines are essential to understand the genetic basis of disease.
• Pluripotent stem cell derived cardiomyocytes can be used to screen for new drugs.
Speakers: Generating Human T cells in Artificial Thymic Organoids Gay Crooks, MBBS, University of California, Los Angeles, USA Learning objectives: • Learn how T cells develop from hematopoietic s...
03:45 PM - 05:00 PM

Strategies for Commercialization Track Session 2 - Regulatory Impact on Global Commercialization of Cell & Gene Therapies

There are numerous cell/gene therapy products (CGTs) with or getting closer to market approval, some of which through utilization of expedited programs at various regulatory Agencies worldwide. We will discuss the potential impact of the strategic approach of regulatory pathways/approvals and their expedited programs across different geographies on global commercialization strategy.

Given the ISCT annual meeting location this year, we will have representation from regulators from Australia, Korea and Japan, and industry representation covering Korea, China, Japan, Australia, Europe and the USA.

Interaction of Regulatory Strategies (Including Conditional Approvals) and Commercialisation Pathways
Dianne Jackson-Matthews, PhD, ERA Consulting, Australia

Development of Cell and Gene Therapy Products: Korea MFDS Regulatory Perspective
So-Young Lee, PhD, Scientific Reviewer, Cell and Gene Therapy Products Division, Natural Institute of Food and Drug Safety Evaluation, Ministry of Food and Drug Safety, Korea

Commercialization Experience of Allogeneic Cell Therapy Product in North Asia - Case Study Across Korea - Japan – China and US
Antonio Lee PhD, MEDIPOST America Inc., United States

Panelists:
Manal Morsy, PhD, MBA, Athersys, United States
Tony Manderson, PhD, TGA, Australia
Yoshiaki Maruyama, PhD, PMDA, Japan
Zonghai Li, MD, PhDCARsgen Therapeutics, China
There are numerous cell/gene therapy products (CGTs) with or getting closer to market approval, some of which through utilization of expedited programs at various regulatory Agencies worldwide. We wi...
03:45 PM - 05:00 PM

Quality and Operations Track Session 2 – Applied Risk Assessment

Risk assessment is a valuable tool that can be used throughout all stages of the product life cycle. This session shall demonstrate via practical examples, the value applied risk assessment can bring to product development, apheresis site selection and qualification and scenarios that can occur during typical cell therapy manufacturing processes.

Chair:
Gerry McKiernan, Cell Therapies Pty Ltd, Australia

Speakers:
A Risk Based Approach to Product Development
Jean Stanton, Janssen Pharmaceuticals, United States

Learning objectives:
• To introduce the concept of risk based approach and its use in product development
• Provide practical application of risk assessment tools through a case study
• Demonstrate how a risk based approach can be used to develop a systematically defined control strategy.

How Risk Management Is Applied to The Cellular Starting Material Used in The Manufacturing of A CAR-T Product
Jeanette Ripper, Novartis Australia, Australia

Learning objectives:
• Understanding that there are many risks associated with the cellular starting material used to manufacture a CAR-T product
• Understanding that there can be considerable challenges in standardising a critical starting material from a large and diverse network of global sites to align with manufacturing process needs
• Understanding that a well-developed risk-based approach is required to ensure that all patient cells received for CAR-T manufacturing meet established acceptance criteria

Reactive Risk Assessment Applied to Issues During Manufacturing
Susan Cheong, Cell Therapies Pty Ltd, Australia
In this session we will step through working examples looking at how we can use various Risk Assessment tools to determine the impact to products and the appropriate next course of action to mitigate reoccurring risks. A variety of Risk Assessment tools shall be demonstrated to show how different techniques can be used depending on the type of event encountered.
Risk assessment is a valuable tool that can be used throughout all stages of the product life cycle. This session shall demonstrate via practical examples, the value applied risk assessment can bring ...
03:45 PM - 05:00 PM

Advanced Practice Professionals (APP track) Session 2 – Novel Therapies for Treating Cancer

Co-Chairs:
Tobi Fisher, MPAS, PA-C, MD Anderson Cancer Center, United States
Megan Cornelison, MS, PA-C, MD Anderson Cancer Center, United States

This session will discuss the current state of art including Chimeric Antigen Receptor T cells and Endogenous T-cells for solid tumors and hematologic malignancies.

Speakers:

CD2-CAR-T cell for GD2-positive Malignancies
Michael Brown, MBBS, PhD, University of South Australia, Australia

Factors impacting initial response and toxicity after CD19 CAR-T cell immunotherapy
Cameron Turtle, MBBS, PhD , Fred Hutchinson Cancer Research Center, United States

Factors Impacting Initial Response and Toxicity After CD19 CAR-T Cell Immunotherapy
Cassian Yee, MD, , MD Anderson Cancer Center, United States
Co-Chairs: Tobi Fisher, MPAS, PA-C, MD Anderson Cancer Center, United States Megan Cornelison, MS, PA-C, MD Anderson Cancer Center, United States This session will discuss the current state of art...
05:15 PM - 06:45 PM

Tackling Cell Therapy Manufacturing Challenges with Closed and Automated Solutions (Corporate Symposium Hosted By Terumo BCT)

Speaker:

Jim Beltzer, MS, PhD, Sr. Manager, Global Strategic Medical Affairs, Cell Therapy Technologies, Terumo BCT, USA

Wenyan Leong, PhD, Cell Processing Specialist, Cell Therapy Technologies, Terumo BCT, Singapore

With the recent FDA approval of the CAR T-cell therapies Kymriah® from Novartis and Yescarta® from Kite Pharma, there has been a renewed interest in bioprocessing and the manufacturing facilities required to meet the commercial demands for these novel cell therapy products. Much of the industry focus has been on the upstream process, with particular emphasis on large-scale cell expansion. In fact, optimization of bioreactor yields often results in downstream process bottlenecks. The challenges of cell therapy process development that arise after the cell expansion stage can include harvesting, washing, cell purification, cell concentration, formulation, fill and finish, and preservation, depending upon the therapy.

A successful commercial production process must be scalable, robust and reproducible and must result in a therapy that is affordable. Compounding the difficulty, many current cell separation technologies are not compatible with large-scale processes. Since the product is the process, manufacturing processes must maintain the functionality and the critical quality attributes of the cellular material throughout.

Learning objectives:
• Gain a greater understanding of the downstream part of the cell processing and its challenges
• Understand the strengths and weaknesses of current approaches
• Discover a new option in the area of final formulation, fill and finish—the Finia® Fill and Finish System from Terumo BCT
• Hands on demonstration of the Finia® Fill and Finish System
Speaker: Jim Beltzer, MS, PhD, Sr. Manager, Global Strategic Medical Affairs, Cell Therapy Technologies, Terumo BCT, USA Wenyan Leong, PhD, Cell Processing Specialist, Cell Therapy Technologies, Ter...
06:00 PM - 06:30 PM

Elevator Pitch Abstract Presentations

Oral Abstract Presentations:

David Curiel (US) – Achievement of Long Term Gene Expression Via Adenoviral Vector-Mediated Delivery Of CRISPR/ Cas9 For In Vivo Editing And Gene Knock-In (Abstract 19)

Mercy Gohil (US) – Myeloid Derived Suppressor Cells (MDSCs) Reduce the Manufacturing Feasibilty Of Gene Modified T Cells. (Abstract 36)

Elise Chong (US) – Impact of CAR T-Cell Product Viability On B-Cell Lymphoid Malignancy Outcomes (Abstract 37)

Sylvie Shen (AU) – Modulation of Anti-Tumour Activity of Ex Vivo Expanded NK and GD2CAR-NK Cells Against Neuroblastoma (Abstract 38)

Rajeswaran Mani (US) – Fc Engineered Anti-CD33mAb Potentiates Cytotoxicity of mbIL-21 Expanded NK-Cells Against Primary AML Pre-Treated with Decitabine (Abstract 39)

Paul Burgoyne (UK) – GMP-Compliant Sorting of Dendritic Cells for CCR7 Expression Improves Therapeutic Efficacy in Cancer by Enhancing Lymph Node Migration and Antigen-Specific T Cell Activation (Abstract 41)

Alasdair Fraser (UK) – Multi-Parameter Flow Cytometric Analysis of Therapeutic EBV-Specific T Cells: Visual Assessment of Product Development and Differentiation Using Dimensionality Reduction with t-SNE. (Abstract 42)

Lorena Braid (CA) – Towards A Consensus Potency Assay for Mesenchymal Stromal Cells: A Matrix Analysis of Cell Source, Donor Variability and Inflammatory Stimuli to Refine Surrogate Markers of Immunomodulation (Abstract 43)

Timothy Wiltshire (US) – Detection of Lentiviral Constructs for Release Testing of CAR- T Cells Using Digital Droplet PCR (Abstract 44)

Catherine Waldby (AU) – Autologous Stem Cell-Based Interventions in Australia: Exploring Patient Experience in Light of Regulatory Exceptionalism (Abstract 45)

Mona Elsemary (AU) – Microfluidic Purification of T Lymphocytes Separated from Blood for Chimeric Antigen Receptor T-cell Manufacturing (Abstract 49)

Susan Prockop (US) – Durable Responses to 3rd Party Viral Specific T Cells Mediated by Different Patterns of Engraftment (Abstract 63)

Dani Offen (IR) – Ectopic Human Muscle Progenitor Cells Expression of Neurotrophic Factors Improves Recovery in a Mouse Model of Sciatic Nerve Injury (Abstract 310)
Oral Abstract Presentations: David Curiel (US) – Achievement of Long Term Gene Expression Via Adenoviral Vector-Mediated Delivery Of CRISPR/ Cas9 For In Vivo Editing And Gene Knock-In (Abstract 19) ...
06:00 PM - 07:30 PM

Poster Session 1

07:30 PM - 09:30 PM

Friday, May 31
06:30 AM - 07:30 AM

ISCT 2019 5K Run

07:30 AM - 08:30 AM

Hot Topic Session 3 – Update on CGT Clinical Trials/Acute CNS Injuries

Speakers:
Translationally Conserved Mechanisms of Benefit Underlying MultiStem Cell Therapy for Treating Acute Neurological Injury
Robert W. Mays, PhD, Athersys, USA

Efficacy and Safety Outcomes in Chronic Traumatic Brain Injury Patients: Phase 2 STEMTRA Trial
Damien Bates, MD, PhD, MBA, SanBio, USA
Speakers: Translationally Conserved Mechanisms of Benefit Underlying MultiStem Cell Therapy for Treating Acute Neurological Injury Robert W. Mays, PhD, Athersys, USA Efficacy and Safety Outcomes in ...
07:30 AM - 08:30 AM

Hot Topic Session 4 – ISCT-KSBMT Joint Session: Advances in Cellular Therapy for Hematologic Disorders

Co-Chairs:
Catherine Bollard, MD, Children's National Medical Center and the George Washington University, United States
Hyoung Jin Kang, MD, PhD Seoul National University School of Medicine, Korea

Speakers:
T cell Therapies for Leukemia/Lymphoma - Beyond CARs
Catherine Bollard, MD, Children's National Medical Center and the George Washington University, United States

Treatment of GVHD With Preconditioned Mesenchymal Stem Cells: From Bench to Bedside
Keon Hee Yoo, MD, PhD, Samsung Medical Center, Korea

Learning Objectives:
•Current challenges in managing steroid-refractory GVHD
•Effects of preconditioning on the immunomodulatory properties of mesenchymal stem cells
•Manufacturing process of GMP-grade Wharton jelly-mesenchymal stem cells for clinical trial
Co-Chairs: Catherine Bollard, MD, Children's National Medical Center and the George Washington University, United States Hyoung Jin Kang, MD, PhD Seoul National University School of Medicine, Korea ...
07:30 AM - 08:30 AM

Avoiding the Potholes Along the Pathway from Process Development To Commercial Cell Therapies (Corporate Tutorial Hosted By Invetech)

*Breakfast available at 07:00 AM

Avoiding the potholes along the pathway from process development to commercial cell therapies

Chair:
Jon Ellis, Manager - Cell Therapy, Invetech, USA

Speakers:
Joanne Kurtzberg, MD Director , Carolinas Cord Blood Bank Duke University Medical Centre, United States

Dawn Driscoll, PhD, MBA, GAICD CEO, Cell Therapies Pty Ltd, Australia

Wen Bo Wang, PhD Senior Vice President, Technical Operations, Fate Therapeutics

The emergence of commercially available cell therapies has been an inflection point for our industry. An initial pathway from Process Development to commercial manufacture has been described, yet there remain many potholes to catch the unwary along the way.
Join us for an insightful discussion on the role that considered automation plays in navigating 3 of the biggest potholes – productivity of human resources, consistency of manufacturing, and access to capital – helping give your therapy a smooth ride to commercial success.
*Breakfast available at 07:00 AM Avoiding the potholes along the pathway from process development to commercial cell therapies Chair: Jon Ellis, Manager - Cell Therapy, Invetech, USA Speakers: Joa...
07:30 AM - 08:30 AM

Strategies for Commercialization Track Session 3 – Capital Without Borders: Raising Money for CGT in the Global Economy

This session will discuss:
- Strategies for biotech access to capital in China
- Improving access to capital in the U.S. for ex-U.S. companies
- Guidelines for Australian biotech companies trying to raise money outside of Australia

Chair:
Patrick Rivers, MBA, Aquilo Capital, USA

Speakers:
Patrick Rivers, MBA, Aquilo Capital, USA
Ting Jia, PhD, Octagon Investments, USA
Michael Sistenich, MSc, Aurenda Partners, AU
This session will discuss: - Strategies for biotech access to capital in China - Improving access to capital in the U.S. for ex-U.S. companies - Guidelines for Australian biotech companies trying to r...
07:30 AM - 08:30 AM

Cytotherapy: A New Era

This session is targeted toward ISCT members, perspective authors, and Early Stage Professionals (ESPs) who are interested in publishing in Cytotherapy and learning more about how the journal serves the society. The session will begin with a brief overview of the journal and its present strengths in the publishing arena. The session will then highlight major changes being instituted at Cytotherapy in 2019 including updates to the journal’s Aims and Scope, cover design, table of contents, Editorial Board, and Senior/Associate/Assistant Editors. Attendees will then be given a brief tutorial about how to successfully prepare a manuscript suitable for publication in Cytotherapy, and a representative from Elsevier will discuss tools offered by the publisher to assist in this process. The session will close with a Q&A period that will include the Senior/Associate Editors and the Elsevier representative and cover all topics related to Cytotherapy.
This session is targeted toward ISCT members, perspective authors, and Early Stage Professionals (ESPs) who are interested in publishing in Cytotherapy and learning more about how the journal serves t...
08:45 AM - 10:15 AM

Plenary Session 3 - ISCT-JSRM Joint Session: Clinical Applications of Human Pluripotent Stem Cells

Co-Chairs:
Ngaire Elwood PhD, BMDI Cord Blood Bank & Murdoch Children's Research Institute, Australia
Yoshiki Sawa MD, Osaka University, Japan

Speakers:

iPS cell-based therapy for Parkinson's disease
Jun Takahashi MD, PhD, Center for iPS Cell Research and Application (CiRA), Kyoto University, Japan

Human induced pluripotent stem cells (iPSCs) can provide a promising source of midbrain dopaminergic (DA) neurons for cell replacement therapy for Parkinson’s disease (PD). Towards clinical application of iPSCs, we have developed a method for 1) scalable DA neuron induction on human laminin fragment and 2) sorting DA progenitor cells using a floor plate marker, CORIN. The grafted CORIN+ cells survived well and functioned as midbrain DA neurons in the 6-OHDA-lesioned rats, and showed minimal risk of tumor formation. In addition, we performed a preclinical study using primate PD models. Regarding efficacy, human iPSC-derived DA progenitor cells survived and functioned as midbrain DA neurons in MPTP-treated monkeys. Regarding safety, cells sorted by CORIN did not form any tumors in the brains for at least two years. Finally, MRI and PET imaging was useful to monitor the survival, expansion and function of the grafted cells as well as immune response by the host brain. Based on these results and pre-clinical studies with a clinical cell line, we have started a clinical trial to treat PD patients at Kyoto University Hospital in Kyoto, Japan in August, 2018.

Retinal Replacement Cell Therapy Using iPS Cells
Masayo Takahashi MD, PhD, Laboratory for Retinal Renegeration, RIKEN Center for Biosystems Dynamics Research, Japan

The first application of iPS-derived cells started in 2014, targeted retinal disease called age-related macular degeneration (AMD). AMD is caused by the senescence of retinal pigment epithelium (RPE), so that we aimed to replace damaged RPE with normal, young RPE made from iPS cells. The grafted autologous RPE cell sheet was not rejected nor made tumor and still functioning after four years. We started the second clinical research using HLA 6 loci homozygous iPSCs from Feb 2017. In this study, HLA 6 loci matched iPSC-derived RPE was transplanted to see if we could manage the immune reaction without systemic immune suppression for the elder patients.
Another cell type in the retina; photoreceptor transplantation is a promising treatment to restore visual function to photoreceptor degenerated retinas such as retinitis pigmentosa. I will talk about the grand design of outer retinal layer replacement therapy.

Addressing the cell scalability challenge head-on: Results of the First Completed Trial of iPSC-Derived MSCs in Steroid-Resistant Acute GvHD
John Rasko AO, BSc(Med), MBBS(Hons), PhD, Royal Prince Alfred Hospital and University of Sydney, Australia

Mesenchymal stromal cells (MSCs) have been widely investigated as a treatment for graft versus host disease (GvHD), but with mixed results. Factors such as MSC donor variability and the effects of prolonged culture expansion may contribute to inconsistent or disappointing outcomes. The novel Cymerus™ manufacturing process facilitates virtually limitless production of well-defined and consistent MSCs from a single human iPSC bank, using clonogenic progenitor-based technology. This avoids both inter-donor variability, batch-to-batch variation and the need for prolonged in vitro expansion of MSCs. We have conducted a multi-centre, open label study of Cymerus MSCs (CYP-001) in adults with steroid-resistant acute GvHD. The primary objective was assessment of safety and tolerability, while the secondary objective was efficacy, based on best responses by Day 28/Day 100 and overall survival. This is the first completed study worldwide with any iPSC-derived product. It has yielded encouraging safety and efficacy data, which support further clinical development of Cymerus iPSC-derived MSCs for GvHD and other indications.
Co-Chairs: Ngaire Elwood PhD, BMDI Cord Blood Bank & Murdoch Children's Research Institute, Australia Yoshiki Sawa MD, Osaka University, Japan Speakers: iPS cell-based therapy for Parkinson's dis...
10:15 AM - 10:30 AM

Corporate Product Theatre

10:15 AM - 10:30 AM

Bridging The Gap From R&D To Commercial Production with Automated, Closed and Scalable Cell Processing Systems (Global Showcase Presentation Hosted by Thermo Fisher Scientific)

Speaker:
Rebecca Lim, PhD, Deputy Centre Head, The Ritchie Centre, Research Group Head, Amnion Cell Biology, Research Fellow, Department of Obstetrics & Gynacology, Monash University, Australia

To ensure a seamless transition from discovery to clinical trials, and ultimately commercial manufacture, Hudson Institute has been exploring the potential of the new Gibco CTS Rotea Counterflow Centrifugation System, a highly reproducible closed cell processing system designed for separation, washing and concentration of both autologous and small-scale allogeneic cell therapy samples. The technology showcase will highlight the unique capability of the Rotea system and the significant impact that it is having on both the manufacturing process and the design and utilization of their translational research facility.

The Monash Health Translation Precinct which co-locates Monash Health (Victoria’s largest public health service provider), Monash University and Hudson Institute is also home to a state of the art translational research facility where clinicians and researchers have access to purpose built facilities and technology platforms which has enabled five firstin-human cell therapy trials in ambulatory and high dependency adults and neonates
Speaker: Rebecca Lim, PhD, Deputy Centre Head, The Ritchie Centre, Research Group Head, Amnion Cell Biology, Research Fellow, Department of Obstetrics & Gynacology, Monash University, Australia To en...
10:15 AM - 10:45 AM

Coffee Break with Exhibits

10:30 AM - 10:45 AM

Corporate Product Theatre Hosted By Rooster Bio

10:30 AM - 10:45 AM

Radically Simplifying Scale-Up and Clinical Translation of hMSCs and Extracellular Vesicles (Global Showcase Presentation Hosted by RoosterBio)

Speaker:
Jon A. Rowley, PhD,Chief Product Officer, RoosterBio Inc, United States

Human Mesenchymal Stem/Stromal Cells (hMSCs) are critical raw materials in numerous therapeutic approaches including cell therapies, cell-based gene therapies, tissue and organ engineering, and exosome and extracellular vesicle-based therapies. RoosterBio has greatly simplified all aspects of product development that include hMSCs including discovery research, product development, manufacturing process development as well as tech transfer and cGMP manufacturing of clinical product. Our hMSC Bioprocess Systems facilitate rapid implementation of fed-batch bioreactor scale-up, and can be directly implemented in large scale bioreactor-based EV production. RoosterBio’s new EV product innovations enable log enhancements in EV lot size and EV productivity, resetting the cost structure of this exciting new technology segment.
Speaker: Jon A. Rowley, PhD,Chief Product Officer, RoosterBio Inc, United States Human Mesenchymal Stem/Stromal Cells (hMSCs) are critical raw materials in numerous therapeutic approaches including ...
10:45 AM - 12:15 PM

Plenary 3 Satellite Session – Production of iPSCs for Clinical Use

Chair: Jun Takahashi, MD, PhD, Professor Center for iPS Cell Research and Application (CiRA), Kyoto University, Japan

Speakers:
Clinical Translation of Induced Pluripotent Stem Cell-Derived Off-the-Shelf Cellular Therapies
Igor Slukvin, MD, PhD, Professor, University of Wisconsin-Madison, United States

Learning Objectives:
•Understand critical quality attributes to clinical grade human pluripotent stem cells (iPSCs) and their derivatives.
•Explain the benefits of using iPSCs as a source of mesenchymal cells for tissue repair and regeneration.
•Discuss the advances and challenges in clinical translation of iPSC-based cellular therapies.

Regenerative Medicine for Spinal Cord Injury Using iPS Cells
Masaya Nakamura, MD, PhD, Professor & Chair Keio University School of Medicine, Department of Orthopedic Surgery, Japan

Learning Objectives:
•To introduce our clinical trial of iPSC derived NCS transplantation for sub-acute SCI
•To introduce our pre-clinical study about chronic SCI
•To introduce our future plan to establish regenerative medicine for chronic SCI

Selected Oral Abstract Presentations

Uma Lakshmipathy (US) – Establishing iPSC Cell Banks Derived Using Reagents and Workflows Optimized for Cell Therapy Manufacturing (Abstract 7)

Jaichandran Sivalingam (SG) Development of A Scalable Agitation Suspension Culture Differentiation Platform for Generating Erythroid Cells from O-Negative Human Induced Pluripotent Stem Cells (Abstract 56)
Chair: Jun Takahashi, MD, PhD, Professor Center for iPS Cell Research and Application (CiRA), Kyoto University, Japan Speakers: Clinical Translation of Induced Pluripotent Stem Cell-Derived Off-the-S...
10:45 AM - 12:15 PM

Plenary 4 Satellite Session – ISCT-Catapult Joint Session: Global Pricing & Reimbursement Strategies

Co-Chairs:
Dawn Driscoll, PhD, MBA, Cell Therapies Pty Ltd, Australia
Jacqueline Barry, PhD, Cell & Gene Therapy Catapult United Kingdom

Over the past 5 years, a number of novel cell and gene therapy products have been approved for commercial use. This has dramatically increased the visibility of CGT products with patients, providers and payers. However, a key component of market access requires the sponsors to achieve adequate and broad reimbursement, and this remains a significant challenge. In this session, experts involved in the reimbursement process will look at the general requirements for achieving listing, discuss their country’s systems for assessing a CGT’s overall value, and pathways for market access.

Speakers:

The Evaluation of Cell and Gene Therapies – Experience from NICE
Nick Crabb, PhD, NICE, United Kingdom

Learning Objectives:
•To outline the challenges of applying health technology assessment to cell and gene therapies
•To highlight the role of managed access arrangements
•To provide case histories from NICE's experience

Pricing, Reimbursement and Market Access of Cellular, and Tissue-based Products in Japan
Yoshimi Yashiro, PhD, Kyoto University, Japan

Learning objectives:
•Explain the public insurance system in Japan
•The Japanese Government uses the costing-up method when determining the national health insurance drug pricing of Cellular, and Tissue-based Products.
•Even if regenerative medical products etc are within the conditional approval period, the patient will not bear the cost burden exceeding the prescribed upper limit.

The Place of Living Therapies in The Funding of Australia’s Health System: Think Global Act Local
Felicity McNeill, PSM, Perspicacité, Australia

The reasoning being the global reference is both the international approach and intersection for Australia (as the panel demonstrates) as well as the move to a global HTA access system for Australia’s reimbursement of services in local state public hospitals and what that means going forward.

CAR T Therapies. How Does Australia’s Health System Meet the Challenge?
Megan Keaney, MBBS, Technology and Assessment Division, Department of Health, Australia
- Like other federations, in Australia, responsibility for health care delivery and funding is shared between the Commonwealth and the states and territories.
- At Commonwealth level , robust health technology assessment ( HTA) is a pre-requisite for funding of technologies and services ,through Commonwealth programs including the PBS and MBS
- At state level the use of the use of HTA is variable but work is being done to move to a nationally cohesive HTA system
- The Medical Services Advisory Committtee’s evaluation of Kymriah is a good example of how HTA can inform national funding decisions, particularly for highly specialised and high cost therapies.
Co-Chairs: Dawn Driscoll, PhD, MBA, Cell Therapies Pty Ltd, Australia Jacqueline Barry, PhD, Cell & Gene Therapy Catapult United Kingdom Over the past 5 years, a number of novel cell and gene thera...
10:45 AM - 12:15 PM

Strategies for Commercialization Track Session 4 – Enabling More Affordable CGT via Manufacturing Efficiencies and Lower COGs

Chair: Ohad Karnieli, PhD, MBA, ATVIO Biotech Ltd, Israel

Speakers:

Jon Rowley (US) – Streamlining hMSC Product & Process Development, Tech Transfer and cGMP Manufacturing to Reduce COGs for Cell, Gene and EV Therapies

Learning objectives:
• The challenges and bottlenecks in scaling hMSC-based product manufacturing
• Media productivity metrics related to both cells and EVs to monitor that correlate directly with COGs
• Opportunities and novel strategies to reduce

Jeff Liter (US) – Effective Cost Reduction Strategies for your CAR-T Therapy

Learning objectives:
• Inform the audience of cost reduction strategies available today
• Show the impact to material cost reduction
• Show the impact to speed to clinic and speed to market

Noam Bercovich (IR) – Why Go for Evolution When Revolution Is an Option? A different approach for CAR-T Therapy

Learning objectives:
• There is so much technology around, it must be integrated with the cell and immunotherapy
• CAR-T therapy can be done more efficiently and economically
• In an area that is considered to be an art automation is the key factor
Chair: Ohad Karnieli, PhD, MBA, ATVIO Biotech Ltd, Israel Speakers: Jon Rowley (US) – Streamlining hMSC Product & Process Development, Tech Transfer and cGMP Manufacturing to Reduce COGs for Cell,...
10:45 AM - 12:15 PM

Quality and Operations Track Session 3 – Technologist Workshop - Global Troubleshooting - Case Studies and Tips

Chair:
Rosemarie Bell, B.App.Sc Micro/Biochem MASM, QIMR Berghofer Medical Research Institute, Australia

An interactive workshop presenting case studies designed to provide a variety of solutions to problems that arise during cell processing and release testing while maintaining compliance from a global perspective. Emergency cryopreservation procedures, isolator environmental control and quality control testing will be discussed.

Speakers:

When Failure is an Option
Heather Garrity, MHA, Dana Farber Cancer Institute, United States

Quality Control Release Testing of Cell Therapy Products
Elise Ross, Cell Therapies Pty Ltd, Australia

Working with Biological Isolators
Gordon McPhee, PhD, Hudson Institute of Medical Research, Australia

A discussion around the operational considerations behind utilising a biological isolator for cell therapy manufacturing; including commissioning, advantages / disadvantages, maintenance and troubleshooting.
Chair: Rosemarie Bell, B.App.Sc Micro/Biochem MASM, QIMR Berghofer Medical Research Institute, Australia An interactive workshop presenting case studies designed to provide a variety of solutions to...
10:45 AM - 12:15 PM

ISCT Presidential Task Force Session – Ethical Issues Across the Cell and Gene Therapy Development Spectrum: From Basic Research to Clinical Translation

Ethical considerations are raised by each phase of the clinical translation pipeline. In this session, four speakers will share their views on issues presented during pre-clinical, clinical and commercialization stages before joining a facilitated panel discussion where current ways in which these issues are, or should be, addressed will be canvassed. This is an opportunity for delegates to raise issues they are contemplating and to learn more about ISCT guidelines.

Wendy Lipworth (AU) – Competing and Conflicting Interests in Clinical Innovation

Learning objectives:
• Appreciate the moral justifications for clinical innovation
• Recognise the need for governance of innovation in the face of competing and conflicting interests

Sarah Chan (UK) – Scientific Freedom and Scientific Responsibility: Ethics in Pre-Clinical Research

Learning objectives:
• Be aware of ethical issues in cell therapy development arising at the pre-clinical stage
• Gain an understanding of how concepts of scientific freedom and responsibility structure the governance of research
• Reflect on social and ethical dimensions of science in the global context

John Rasko (AU) – Ethical Issues in Commercializing Cell Therapies

Bruce Levine (US) – Ethical Issues in Cell Therapy Clinical Trials
Ethical considerations are raised by each phase of the clinical translation pipeline. In this session, four speakers will share their views on issues presented during pre-clinical, clinical and commer...
12:15 PM - 01:45 PM