Wednesday, May 29
10:00 AM - 10:15 AM

10:15 AM - 11:45 AM

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

Speakers
  • Joanne Kurtzberg MD, Director, Carolinas Cord Blood Bank, Duke University Medical Center (Speaker)
10:15 AM - 11:45 AM

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

Speakers
  • Donald G. Phinney PhD, Professor, The Scripps Research Institute, Scripps Florida (Chairman)
11:45 AM - 01:00 PM

02:30 PM - 02:45 PM

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

Corporate Product Theatre Opening Hosted by CCRM

Thursday, May 30
07:30 AM - 08:30 AM

Hot Topic Session 1 - Bioengineering of the GI Tract

Speakers
  • Tracy Grikscheit MD, Attending Physician, Children's Hospital of Los Angeles (Speaker)
  • Giuseppe Orlando MD, PhD, Assistant Professor, Wake Forest University Baptist Medical Center (Speaker)
  • Rachele Ciccocioppo MD, Associate Professor in Gastroenterology, AOUI Policlinico G.B. Rossi & University of Verona School of Medicine (Chairman)
09:00 AM - 10:30 AM

Presidential Plenary - CAR Ts Beyond B Cells

CART cells for acute myeloid leukemia
Saar Gill MD, PhD, University of Pennsylvania, USA
In this presentation, the reasons CAR T cell therapy for myeloid malignancies has lagged behind that for B-lymphoid malignancies will be reviewed and several potential ways to bring this potent therapeutic modality to patients will be outlined.

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
CART cells for acute myeloid leukemia Saar Gill MD, PhD, University of Pennsylvania, USA In this presentation, the reasons CAR T cell therapy for myeloid malignancies has lagged behind that for B-ly...
10:30 AM - 10:45 AM

Corporate Product Theatre Hosted By GE Healthcare

10:30 AM - 11:00 AM

Coffee Break with Exhibits

10:45 AM - 11:00 AM

Corporate Product Theatre Hosted By Sartorius Stedim Biotech

11:00 AM - 12:15 PM

Plenary 1 Satellite - Immunotherapy Oral Abstract Presentations I

Selected Oral Abstract Presentations
Selected Oral Abstract Presentations
Speakers
  • Saar Gill MD, PhD, Assistant Professor, University of Pennsylvania (Chairman)
11:00 AM - 12:15 PM

Plenary 2 Satellite - Enabling Technologies

Speakers
  • Tracy Grikscheit MD, Attending Physician, Children's Hospital of Los Angeles (Chairman)
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.
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 ...
Speakers
  • Heidi Hagen MS, MBA, Co-Founder & Chief Strategy Officer, Vineti, Inc (Speaker)
  • Kilian Kelly PhD, Vice President, Product Development, Cynata Therapeutics (Speaker)
  • Andrew McDonald, Director, Australia & New Zealand, World Courier (Speaker)
  • See More..
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
Development of a Cryopreservation Standard to Speed Cell Therapy/Regenerative Medicine Product Development
Brian J. Hawkins, PhD Pluristyx Inc, United States
ISO Standards for Ancillary Materials and Transportation
Claudia Zylberberg, PhD Akron Biotech, 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 State...
Speakers
  • Claudia Zylberberg PhD, CEO & Founder, Akron Biotech (Speaker)
  • Sowmya Viswanathan PhD, Affiliate Scientist, University Health Network Cell Therapy Program (Chairman)
11:00 AM - 12:15 PM

Advanced Practice Professionals Session 1 - Cord-Blood Derived Therapies for Regenerative Medicine & Transplantation

Speakers:

Juliet Barker, MBBS, Memorial Sloan Kettering Cancer Center, United States
Ngaire Elwood, PhD, Murdoch Children's Research Institute, Australia
Speakers: Juliet Barker, MBBS, Memorial Sloan Kettering Cancer Center, United States Ngaire Elwood, PhD, Murdoch Children's Research Institute, Australia
Speakers
  • Juliet Barker MBBS, FRACP, Attending Physician, Memorial Sloan Kettering Cancer Center (Speaker)
  • Ngaire Elwood PhD, Director, BMDI Cord Blood Bank & Head Cord Blood Stem Cell Research Program, Murdoch Children's Research Institute (Speaker)
12:15 PM - 01:45 PM

Lunch with Exhibits

12:30 PM - 12:45 PM

Corporate Product Theatre Hosted By Australian Red Cross Blood Service

12:45 PM - 01:00 PM

Corporate Product Theatre

01:00 PM - 01:15 PM

Corporate Product Theatre Hosted By AventaCell Biomedical

01:15 PM - 01:30 PM

Corporate Product Theatre Hosted By Merck

01:45 PM - 03:15 PM

Plenary Session 2 - Tissue Engineering

Co-Chairs:
Oscar K. Lee MD, MSc, PhD, National Yang-Ming University, Taiwan
Janet Macpherson PhD, GE Healthcare Life Sciences, Australia
Speakers:
Melissa Little PhD, Murdoch Children's Research Institute, Australia

Clinical Application of Engineering Cell Sheet for Cardiac Repair
Yoshiki Sawa MD, 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.

Tracy Grikscheit MD, Children's Hospital of Los Angeles, USA
Co-Chairs: Oscar K. Lee MD, MSc, PhD, National Yang-Ming University, Taiwan Janet Macpherson PhD, GE Healthcare Life Sciences, Australia Speakers: Melissa Little PhD, Murdoch Children's Research Insti...
03:15 PM - 03:45 PM

Corporate Product Theatre Hosted By Pall

03:15 PM - 03:45 PM

Coffee Break with Exhibits

03:45 PM - 05:00 PM

Plenary 1 Satellite - Immunotherapy Oral Abstract Presentations II

Selected Oral Abstract Presentations
Selected Oral Abstract Presentations
Speakers
  • Franco Locatelli MD, PhD, Full Professor of Pediatrics, University of Pavia and IRCCS Ospedale Pediatrico Bambino Gesù (Chairman)
03:45 PM - 05:00 PM

Plenary 2 Satellite - Organoids for Regenerative Medicine

Speakers:

Gay Crooks, MBBS, University of California, Los Angeles, United States
David Elliott, PhD , Murdoch Children's Research Institute, Australia
Speakers: Gay Crooks, MBBS, University of California, Los Angeles, United States David Elliott, PhD , Murdoch Children's Research Institute, Australia
Speakers
  • Gay Crooks MBBS, Professor, University of California Los Angeles (Speaker)
  • David Elliott PhD, Team Leader, Murdoch Children's Research Institute (Speaker)
  • Melissa Little PhD, Theme Director of Cell Biology, Murdoch Children's Research Institute (Chairman)
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 China, Australia 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

New updates of the regulatory requirements for cell therapy products in China
Bao-Zhu Yuan MD, PhD, NMPA, China

Commercialization experience of allogeneic cell therapy product in North Asia - case study across Korea - Japan – China and US
Antonio Lee PhD, MEDIPOST Co, Ltd, Korea
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...
Speakers
  • Dianne Jackson-Matthews PhD, Chief Scientific Officer, ERA Consulting (Speaker)
  • Antonio Lee PhD, CEO and Managing Director, MEDIPOST Co, Ltd (Speaker)
  • Bao-Zhu Yuan MD, PhD, Department Chief, NMPA (Speaker)
  • See More..
03:45 PM - 05:00 PM

Quality and Operations Track Session 2 - Applied Risk Assessment

Chair:
Gerry McKiernan, Cell Therapies PTY, Australia

Speakers:
Susan Cheong, Cell Therapies PTY, Australia
Jean Stanton, Janssen Pharmaceuticals, United States
Jeanette Ripper, Novartis Oncology, Australia
Chair: Gerry McKiernan, Cell Therapies PTY, Australia Speakers: Susan Cheong, Cell Therapies PTY, Australia Jean Stanton, Janssen Pharmaceuticals, United States Jeanette Ripper, Novartis Oncology,...
Speakers
  • Jean Stanton, Director, CAR-T Apheresis Site Liaisons, Janssen Pharmaceuticals (Speaker)
03:45 PM - 05:00 PM

Advanced Practice Professionals Session 2 - Novel Therapies for Treating Cancer

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

Cassian Yee, MD, , MD Anderson Cancer Center, United States
Speakers: Factors impacting initial response and toxicity after CD19 CAR-T cell immunotherapy Cameron Turtle, MBBS, PhD , Fred Hutchinson Cancer Research Center, United States Cassian Yee, MD, , MD ...
Speakers
  • Michael Brown MBBS, PhD, Professor, University of South Australia (Speaker)
  • Cameron Turtle MBBS, PhD, Associate Member and Attending Physician, Fred Hutchinson Cancer Research Center (Speaker)
  • Cassian Yee MD, Professor, MD Anderson Cancer Center (Speaker)
06:00 PM - 07:30 PM

Poster Session 1

07:00 PM - 09:00 PM

07:30 PM - 09:30 PM

ISCT Industry Networking Event

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

07:30 AM - 08:30 AM

Hot Topic Session 3 - Update on CGT Clinical Trials for Stroke/Acute CNS

Speakers
  • Charles S. Cox MD, Professor of Pediatric Surgery and George and Cynthia Mitchell Distinguished Chair in Neuroscience, University of Texas Medical School (Chairman)
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
Jong-Ho Won, MD, PhD, Soonchunhyang University Hospital, Korea

Speakers:
Catherine Bollard, MD, Children's National Medical Center and the George Washington University, United States

Mesenchymal stem cell therapy for GVHD
Keon Hee Yoo, MD, PhD, Samsung Medical Center, Korea
Co-Chairs: Catherine Bollard, MD, Children's National Medical Center and the George Washington University, United States Jong-Ho Won, MD, PhD, Soonchunhyang University Hospital, Korea Speakers: Cat...
Speakers
  • Catherine Bollard MD, Professor of Pediatrics and of Microbiology, Immunology and Tropical Medicine, Children's National Medical Center (Speaker)
  • Keon Hee Yoo MD, PhD, Professor, Samsung Medical Center (Speaker)
  • Catherine Bollard MD, Professor of Pediatrics and of Microbiology, Immunology and Tropical Medicine, Children's National Medical Center (Chairman)
07:30 AM - 08:30 AM

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
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 rais...
Speakers
  • Patrick Rivers MBA, Senior Vice-President, Research, Aquilo Capital (Chairman)
08:45 AM - 10:15 AM

Plenary Session 3 - ISCT-JSRM Joint Session: Clinical Applications of hPSCs

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.

John Rasko AO, BSc(Med), MBBS(Hons), PhD, Royal Prince Alfred Hospital and University of Sydney, Australia
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 dise...
10:15 AM - 10:30 AM

Corporate Product Theatre

10:15 AM - 10:45 AM

Coffee Break with Exhibits

10:30 AM - 10:45 AM

Corporate Product Theatre Hosted By Rooster Bio

10:45 AM - 12:15 PM

Plenary 3 Satellite - Production of iPSCs for Clinical Use

Speakers
  • Masaya Nakamura MD, PhD, Professor & Chair, Keio University School of Medicine, Department of Orthopedic Surgery (Speaker)
  • Jun Takahashi MD, PhD, Professor, Center for iPS Cell Research and Application (CiRA), Kyoto University (Chairman)
10:45 AM - 12:15 PM

Plenary 4 Satellite - Global Pricing & Reimbursement Strategies

Speakers
  • Nick Crabb PhD, Program Director - Scientific Affairs, NICE (Speaker)
  • Felicity McNeill PSM, Managing Director, Perspicacité (Speaker)
  • Jacqueline Barry PhD, Chief Clinical Officer, Cell and Gene Therapy Catapult (Chairman)
  • See More..
10:45 AM - 12:15 PM

Quality and Operations Track Session 3 - Technologist Workshop

Chair:
Rosemarie Bell, B.App.Sc Micro/Biochem MASM, QIMR Berghofer Medical Research Institute, Australia
Speakers:
Heather Garrity, MHA, Dana Farber Cancer Institute, United States
Elise Ross, Peter MacCallum Cancer Centre, Australia
Gordon McPhee, PhD, Hudson Institute of Medical Research, Australia
Chair: Rosemarie Bell, B.App.Sc Micro/Biochem MASM, QIMR Berghofer Medical Research Institute, Australia Speakers: Heather Garrity, MHA, Dana Farber Cancer Institute, United States Elise Ross, Peter...
12:15 PM - 01:45 PM

Lunch with Exhibits

12:30 PM - 12:45 PM

Corporate Product Theatre Hosted By Cynata Therapeutics

12:45 PM - 01:00 PM

Corporate Product Theatre Hosted By Alliance fro Cell and Gene Therapies

01:00 PM - 01:15 PM

Corporate Product Theatre Hosted by Sartorius Stedim Biotech

01:15 PM - 01:30 PM

Corporate Product Theatre Hosted By FUJIFILM

PRIME XV T cell media solutions for T cell based immunotherapy cell culture processes
Vanda S. Lopes, PhD, FUJIFILM Irvine Scientific
Cell therapy is a growing area that focuses on the application of cells as the therapeutic product, and where cell culture is the manufacturing process. Currently the main challenges in this approach reside in the generation of enough cell numbers, with consistent clinical quality. While this may seem rather straightforward, cell culture is a process that involves a large number of interlinked variables, as cell quality and culture media. The combination of cell population with the exact culture process tends to unique for each laboratory, and thus a basal expansion media is seen as a generic reagent that may benefit from customization to be optimal for each setup.
A move towards a consistent manufacturing process requires the removal of undefined components, and thus FUJIFILM Irvine Scientific has two PRIME XV T cell media options available for expansion of T cells: PRIME XV T Cell XSFM and PRIME XV T Cell CDM. We now took media optimization to the next level and developed a serum-replacement supplement (PRIME XV Immune Serum Replacement) that allows the generic basal media to be effective in a wider range of T cell culture setups, while maintaining the benefits of a xeno, serum-free culture system. In here, we will discuss the impact of culture conditions on media performance and the benefits of a serum replacement supplement.
PRIME XV T cell media solutions for T cell based immunotherapy cell culture processes Vanda S. Lopes, PhD, FUJIFILM Irvine Scientific Cell therapy is a growing area that focuses on the application of ...
01:45 PM - 03:15 PM

Plenary Session 4 - Commercial Strategies for Expanding Global CGT Access

Chair:
Miguel Forte, MD, PhD, Zelluna Immunotherapy, Norway
Speaker:
Bruce Levine, PhD, University of Pennsylvania, United States
Silviu Itescu, MBBS, Mesoblast, Australia
Tony Liu, CBMG, China
Chair: Miguel Forte, MD, PhD, Zelluna Immunotherapy, Norway Speaker: Bruce Levine, PhD, University of Pennsylvania, United States Silviu Itescu, MBBS, Mesoblast, Australia Tony Liu, CBMG, China
03:15 PM - 03:30 PM

Corporate Product Theatre

03:15 PM - 03:45 PM

Coffee Break with Exhibits

03:30 PM - 03:45 PM

Corporate Product Theatre

03:45 PM - 05:00 PM

Plenary 3 Satellite - Regulatory & Quality Considerations for iPSC Banking

Speakers
  • Masayo Takahashi MD, PhD, Project Leader, Laboratory for Retinal Renegeration, RIKEN Center for Biosystems Dynamics Research, Japan (Chairman)
03:45 PM - 05:00 PM

Plenary 4 Satellite - Understanding the Challenges to Harmonizing CGT Clinical Trials across Multiple Countries

The development of cell and gene therapy products (CGTs) is occurring across multiple indications and countries. This session will present clinical strategies undertaken by companies based in China, Korea and Australia as they move their CGTs forward into multiple geographic regions including Asia, Europe and North America. Speakers will discuss approaches to clinical development with different countries from IND authorization to market approval.

Development of GPC3-specific CAR T cells for the treatment of patients with GPC3-positive tumors
Zonghai, Li, MD, PhD, CARsgen Therapeutics, China

We and others demonstrated that GPC3 is a cancer-selective target. And we first demonstrated that GPC3 chimeric antigen receptor (CAR) T cells are highly effective in the eradication of hepatocellular carcinoma as well as squamous lung cancer in mouse models. In 2015, we initiated a first-in-human exploratory clinical study on GPC3 CAR T for the treatment of patients with hepatocellular carcinoma. Considering its safety and promising efficacy, we submitted IND to China FDA (now called NMPA). And in January of 2019, we got IND clearance on this program, which is the first IND approval of CAR T cells against solid tumor in China.

Globalization of a Game Changer - the Invossa Story
Soohyun Lew, MD, , Kolon Tissuegene, Korea
The development of cell and gene therapy products (CGTs) is occurring across multiple indications and countries. This session will present clinical strategies undertaken by companies based in China, ...
Speakers
  • Soohyun Lew MD, Vice President/Medical Director, Kolon Tissuegen (Speaker)
  • Zonghai Li MD, PhD, Executive Chairman, Chief Executive Officer, Chief Scientific Officer and Founder, CARsgen Therapeutics (Speaker)
  • Anthony Ting PhD, Vice President of Regenerative Medicine and Head of Cardiopulmonary Programs, Athersys, Inc. (Chairman)
03:45 PM - 05:00 PM

Quality and Operations Track Session 4 - How Do You Determine Stability of Cell Based Therapies?

Chair and Speaker:
Cryopreservation and stability program for hematopoietic stem/progenitor cell grafts
Nadim Mahmud, MD, PhD, University of Illinois College of Medicine, United States

Speakers:
Stability testing utilising intracellular cytokine staining and degranulation assays
Melissa Rist, PhD, QIMR Berghofer Medical Research Institute, Australia
Cryopreservation and stability program involving non-Hematopoietic cells
Michael Bellio, PhD University of Miami, United States
Chair and Speaker: Cryopreservation and stability program for hematopoietic stem/progenitor cell grafts Nadim Mahmud, MD, PhD, University of Illinois College of Medicine, United States Speakers: S...
Speakers
  • Nadim Mahmud MD, PhD, Professor of Medicine, University of Illinois College of Medicine (Speaker)
03:45 PM - 05:00 PM

Early Stage Professionals Session - Training the Next Generation of Cell & Gene Therapy Professionals

Speakers
  • David Bishop MBBS, Haematologist, Westmead Institute for Medical Research (Speaker)
  • Zlatibor Velickovic PhD, Production Manager, Royal Prince Alfred Hospital (Speaker)
  • Emily Hopewell PhD, Director of Cell & Gene Therapy Manufacturing, Indiana University (Chairman)
03:45 PM - 05:00 PM

Strategies for Commercialization Track Session 5 - Supply Chain Consistency

Speakers
  • Dominic Clarke PhD, Global Head of Cell Therapy, HemaCare (Speaker)
  • Claudia Zylberberg PhD, CEO & Founder, Akron Biotech (Speaker)
  • Dolores Baksh PhD, Innovation Leader, Cell & Gene Therapies, GE Healthcare (Chairman)
05:00 PM - 06:30 PM

Poster Session 2

07:00 PM

ISCT 2019 Gala

Saturday, June 1
08:00 AM - 09:00 AM

Hot Topic Session 5 - Advances in Cord Blood Transplantation for CGT

Speakers
  • Juliet Barker MBBS, FRACP, Attending Physician, Memorial Sloan Kettering Cancer Center (Speaker)
  • Joanne Kurtzberg MD, Director, Carolinas Cord Blood Bank, Duke University Medical Center (Speaker)
  • Ngaire Elwood PhD, Director, BMDI Cord Blood Bank & Head Cord Blood Stem Cell Research Program, Murdoch Children's Research Institute (Chairman)
08:00 AM - 09:00 AM

08:00 AM - 09:00 AM

Quality and Operations Track Session 5 - Gene Therapy Vector Quality

Speaker:
Shirley Bartido, PhD, Cellectis, United States
Speaker: Shirley Bartido, PhD, Cellectis, United States
09:15 AM - 10:45 AM

Plenary Session 5 - Immune Monitoring and Tumor Escape

Co-Chairs:
Patrick Hanley, PhD , Children's National Medical Center, USA

This session focuses on immune monitoring of novel therapies, including using novel biomarkers, factors that impact duration of response, and bioinformatic approaches to mass cytometry and the use of t-SNE and PCA. The session will also evaluate tumor escape in a solid tumor model and the role of TNF in resistance to immunotherapy

Factors impacting duration of response in adults treated with CD19 CAR-T cell immunotherapy
Cameron Turtle, MBBS, PhD, Fred Hutchinson Cancer Research Center, USA

Lymphodepletion chemotherapy followed by infusion of T cells that are genetically modified to express a chimeric antigen receptor (CAR) targeted to CD19 is a novel therapy for patients with relapsed and/or refractory B cell acute lymphoblastic leukemia, non-Hodgkin lymphoma, and chronic lymphocytic leukemia. We have investigated factors that impact the duration of response in adults receiving CD19 CAR-T cell therapy.

TNF in anti-tumour immunity and resistance to immunotherapy
Jane Oliaro, BSc (Hons), PhD, Peter MacCallum Cancer Centre, Australia

Immunotherapies that enhance cytotoxic T cell activity against tumour cells have revolutionised outcomes for cancer patients. However patient responses vary widely, so there is considerable interest in understanding how tumours evade this form of therapy. To investigate this, we carried out a series of CRISPR screens to identify mechanisms of tumour immune evasion from T cell killing. We found that deletion of key genes within the TNF signalling, IFN-gamma signalling, and antigen presentation pathways provided protection of tumour cells from T cell killing, and blunted anti-tumour immune responses in vivo. Our results also highlighted a role for TNF-mediated bystander killing as a potent T cell effector mechanism that can be enhanced by a class of drugs, called smac-mimetics, that inhibit IAPs and can sensitise tumour cells to TNF-induced cell death. Indeed, our studies showed that the smac-mimetic, birinapant, significantly enhanced tumour cell death in the presence of T cells, an effect that was amplified upon checkpoint blockade. Furthermore, birinapant significantly enhanced CAR T cell therapy in a solid tumour setting. Taken together, we identify T cell-derived TNF as a potent anti-tumour effector mechanism that can be enhanced with birinapant, and an opportunity for combination therapy through co-inhibition of immune checkpoints.

In vivo dynamics of adoptively transferred T cells - evidence from human trials
Emily Blyth, B.Med(Hons), FRACP, FRCPA, PhD, Westmead Hospital, Australia

Adoptive T cell therapy with pathogen specific T cells for the treatment of immune deficiency following allogeneic haemopoietic stem cell transplant (HSCT) has shown clinical efficacy with little toxicity. Graft manipulation strategies and adoptive cell transfer after HSCT are ever more complex but the biological effects are not well understood. Small patient numbers and complex clinical features pose significant challenges.
We have employed high dimensional mass cytometry and a bioinformatics pipeline to study immune reconstitution in recipients of allogeneic HSCT with and without adoptive T cell therapy using a 37 marker panel producing 75 gated canonical cell subsets per patient sample. We have used bioinformatics tools including t-stochastic neighbour embedding (t-SNE), principle component analysis (PCA) and unsupervised consensus clustering algorithm SC3 to analyse this large dataset. Immunological profiles have been identified that are influenced by clinical parameters such as transplant conditioning, donor source, T cell depletion and post-transplant events, in particular, CMV reactivation, adoptive T cell transfer of pathogen specific T cells and time post-transplant. Immune recovery within each individual can be visualised in detail using spanning-tree progressive algorithm of density normalised events (SPADE) or visualisation of t-SNE (ViSNE).
In order to assess the fate of transferred clones, we have used deep sequencing of TCR- in patients on adoptive T cell therapy trials. Notable differences were seen in TCR diversity and specificity pre-and post-infusion in patients receiving donor-derived cells, and adoptively transferred cells were seen to rapidly engraft. In contrast, patients who received third party T cells did not demonstrate significant engraftment of third-party cells, despite clinical recovery from the targeted infection and immune recovery.
The system level immunological changes form cell therapy interventions can be understood using mass cytometry immune profiling and TCR clone tracking. This technology has numerous potential applications in cancer immunotherapy.
Co-Chairs: Patrick Hanley, PhD , Children's National Medical Center, USA This session focuses on immune monitoring of novel therapies, including using novel biomarkers, factors that impact duration o...
10:45 AM - 11:00 AM

11:00 AM - 12:00 PM

Plenary 6 Satellite - Challenges in Translating MSC EVs into the Clinic

Speakers
  • Bernd Giebel PhD, Senior Scientist, University Hospital Essen, Institute for Transfusion Medicine (Speaker)
  • Sai-Kiang Lim, Research Director, Institute of Medical Biology (IMB) (Chairman)
11:00 AM - 12:00 PM

Strategies for Commercialization Track Session 6 - Logistics of Adoption of Cell & Gene Therapies - A Center and Patient-Focused Perspective

After the initial success of adoptive immunotherapy with chimeric antigen receptor T (CAR-T) cells in clinical trials leading to the market approval of two such products in the US followed by the EU, many medical centers have been tasked with making these therapies available to their patients. It is not an easy process to implement cell and gene therapy based therapies in medical centers focused on patient care and not clinical trials; not all centers are appropriately equipped and staffed to carry out the application of these therapies. In addition, many patients and patient advocates already are demanding to make such treatments available at a larger scale, and in an affordable manner. An international effort is currently underway to provide these therapies to patients in need. It will be reviewed how these therapies were made available in a major US center, and we will also provide an update on implementation of these therapies in Australia.
After the initial success of adoptive immunotherapy with chimeric antigen receptor T (CAR-T) cells in clinical trials leading to the market approval of two such products in the US followed by the EU, ...
Speakers
  • Dominic Wall PhD, Director of Pathology Operations, Peter MacCallum Cancer Center (Speaker)
  • Gerhard Bauer PhD, Director GMP Facility, University of California Davis Institute for Regenerative Cures (Chairman)
11:00 AM - 12:00 PM

Quality and Operations Track Session 6 - Qualification of Computer Systems

Chair:
Annette Trickett, PhD, MAppSc, FIBMS, Prince of Wales Hospital, Australia

Speaker:
Guy Klamer, PhD, Sydney Cord Blood Bank, Australia
Chair: Annette Trickett, PhD, MAppSc, FIBMS, Prince of Wales Hospital, Australia Speaker: Guy Klamer, PhD, Sydney Cord Blood Bank, Australia
12:00 PM - 01:30 PM

12:15 PM - 01:15 PM

ISCT Annual General Business Meeting

01:30 PM - 03:00 PM

Plenary Session 6 - MSCs Towards the Clinic

Chair:
Donald G. Phinney PhD, The Scripps Research Institute, Scripps Florida, USA
Speakers:
Eleuterio Lombardo PhD, TiGenix (Takeda Group), Spain
Douglas Losordo MD , Caladrius Biosciences, USA
Joanne Kurtzberg MD, , Duke University Medical Center, USA
Chair: Donald G. Phinney PhD, The Scripps Research Institute, Scripps Florida, USA Speakers: Eleuterio Lombardo PhD, TiGenix (Takeda Group), Spain Douglas Losordo MD , Caladrius Biosciences, USA Joan...
03:00 PM - 03:15 PM

03:15 PM - 04:30 PM

Plenary 5 Satellite - ISCT-SITC Joint Session: Mechanisms of Tumor Resistance to Targeted Cellular Cancer Therapy‎

Speakers
  • Timothy Cripe MD, PhD, Division Chief, Nation Wide Children's Hospital (Speaker)
  • Hideho Okada MD, PhD, Professor, UCSF (Speaker)
  • Cassian Yee MD, Professor, MD Anderson Cancer Center (Chairman)
03:15 PM - 04:30 PM

Plenary 6 Satellite - MSCs as Therapeutic Targets

Speakers
  • Louise Purton PhD, Associate Director and Lab Head, St. Vincent's Institute (Speaker)
  • Eleuterio Lombardo PhD, Scientific Director, TiGenix (Takeda Group) (Chairman)
03:15 PM - 04:30 PM

Strategies for Commercialization Track Session 7 - Manufacturing for Globalization

Speakers
  • Kim Raineri MBA, Vice President of Operations, Nikon CeLL Innovation Co., Ltd. (Speaker)
  • Michael May PhD, President and CEO, CCRM (Chairman)
03:15 PM - 04:30 PM

Quality and Operations Track Session 7 - Manufacturing of Customary and Complex Therapies: Techniques, challenges, and tricks

Chair:
Aisha Khan, MSc, MBA, Interdisciplinary Stem Cell Institute, University of Miami, United States

Speakers:
Joseph Schwartz, MD, MPH Columbia University Medical Center, United States
Joseph Gold, PhD, City of Hope National Medical Center, United States
Paul Eldridge, PhD, University of North Carolina-Chapel Hill, United States
Chair: Aisha Khan, MSc, MBA, Interdisciplinary Stem Cell Institute, University of Miami, United States Speakers: Joseph Schwartz, MD, MPH Columbia University Medical Center, United States Joseph Go...