Program
ISCT 2021 New Orleans VIRTUAL focuses on six key topic areas in translational science, as well as key issues in regulation and industry
SCIENTIFIC PROGRAM
TRACK I
Mesenchymal Stromal Cells
TRACK II
Commercialization
TRACK III
Exosomes
TRACK IV
Stem Cell Engineering & Bone Marrow Transplantation
TRACK V
Tissue Engineering & Regenerative Medicine
TRACK VI
Immuno & Gene Therapies
REGULATORY PROGRAM
GRP Workshop • Regulatory Sessions
INDUSTRY PROGRAM
Corporate Symposia • Roundtables • Global Showcase • Tutorials
Use the drop-down menu below to select a program track to follow.
You may also search by keywords by using the search box below.
- Filter by Topic, Schedule, and More
- Plenary
- Track I - Mesenchymal Stromal Cells
- Track II - Commercialization
- Track III - Exosomes
- Track IV - Stem Cell Engineering & BMT
- Track V - Tissue Engineering & Regenerative Medicine
- Track VI - Immuno & Gene Therapies
- Regulatory Sessions
- Hot Topics Industry Roundtable
- Corporate Symposia
- Corporate Tutorials
- Global Showcase
- Pre-Recorded Sessions
COVID-19: Understanding the Path Forward for Mesenchymal Stromal Cell Therapies
MAY 26 - 08:00 EDT
In 2020, MSC therapies offered ‘a new hope’ for treating COVID-19. At the ISCT 2020 Virtual Meeting, ISCT explored the scientific rationale and growing list of MSC trials for COVID-19 induced acute respiratory distress syndrome (ARDS). One year later, our panelists return to provide an update on the very latest results from ongoing MSC trials for COVID-19, and to discuss how we can apply this new knowledge to address the ongoing hurdles to MSC therapeutic development.
Presenters:
Jacques Galipeau, MD
University of Wisconsin-Madison
USA
Cells for Cells – REGENERO
Chile
Federal University of Rio de Janeiro
Brazil
Bone Therapeutics
USA
Abstract Presentation
Tomer Bronshtein, PhD
Bonus BioGroup Ltd.
Israel
Abstract Presentation
Duncan Stewart, MD
Ottawa Hospital Research Institute
Canada
Abstract Presentation
Rita Barcia, PhD
Sentien Biotechnologies
USA
Abstract Presentation
Silviu Itescu, MBBS, FRACP
Mesoblast
Australia
Integrating Science, Regulatory Oversight, and Commercialization for the New Wave of Cell & Gene Therapies
Program Track Sponsored by: | ![]() ![]() |
MAY 26 - 13:30 EDT
Forecasts demonstrate that approvals-wise, we are looking at 10-20 items being considered per year by 2025. For some time now, we have been lining up to build the research and the knowledge needed for cell and gene therapies to flourish... The questions we can ask now pivot towards how approval should be approached, how reimbursement might look, how patients can get access to therapies, and so on.
Presenters:
Bruce Levine, PhD
University of Pennsylvania
USA
Shabnum Patel, PhD
Stanford University
USA
Peter Marks, MD, PhD
FDA Center for Biologics Evaluation and Research
USA
Allogene Therapeutics
USA
Stanford University
USA
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"First in Man" Exosome Trials
MAY 27 - 08:00 EDT
Human trials of exosome therapeutics mark the arrival of true clinical translation. Understanding manufacturing and regulatory challenges is critical to ensure a smooth translational path forward. The panel considers the translation of current preclinical data and lessons learned from research to date to examine where consensus is needed, and how it can be developed.
Presenters:
Sai-Kiang Lim, PhD
Institute of Molecular & Cell Biology
Singapore
Maurizio Muraca, MD, PhD
University of Padova
Italy
Eva Rohde, MD
Paracelsus Medical University
Austria
University of Pennsylvania
USA
University of Modena and Reggio Emilia
Italy
BMT vs Gene Therapy vs No Cell Therapy: Accessibility, Cost, and Outcomes
May 27 - 13:30 EDT
The panel begins with clinical updates exploring the latest data in gene therapy and alternative donor BMT for treating hemoglobinopathies, followed by a moderated debate with leading experts to understand accessibility, cost, endpoints, and outcomes of the currently available therapies for thalassemia and sickle cell disease.
Presenters:
Jaap Jan Boelens, MD, PhD
Memorial Sloan Kettering
USA
Sandeep Soni, MD
CRISPR Therapeutics
USA
Ann & Robert H. Lurie Children's Hospital of Chicago
United States
Ramathibodi Hospital, Mahidol University
Thailand
University Children's Hospital Regensburg
Germany
Stanford Medicine
USA
The Children's Hospital at Montefiore
USA
Charting the Future - Safe and Effective Translation in Tissue Engineering and Regenerative Medicine
MAY 28 - 08:00 EDT
Successfully translating tissue engineered and regenerative medicine products requires orchestration of numerous disciplines to realize the potential to repair, replace, and regenerate organs and tissues. This panel examines the state of the art, and key lessons from, stem cell therapy and 3d printing research, proposing a path forwards towards safe translation.
Presenters:
Ranieri Cancedda, MD
Università degli Studi di Genova
Italy
Giuseppe Orlando, MD, PhD
Wake Forest University
USA
Wake Forest Institute for Regenerative Medicine
USA
University of Modena and Reggio Emilia
Italy
Chinese University of Hong Kong
Hong Kong
Debating Applications of CAR-T/NK cells: Perspectives on Autologous versus Allogeneic Therapies
Program Track Sponsored by: | ![]() ![]() |
MAY 28 - 13:30 EDT
Leading experts in the field outline the benefits and challenges of autologous versus allogeneic sources of immune effector cells, drawing on their experiences in driving novel technologies within each setting. The panel will discuss how progress is contingent on addressing issues related to manufacturing, efficacy, regulatory approvals, cost, patient access, pricing, and
availability of therapies to underserved populations. Through this discussion, the panel aims to build consensus that supports decisions for the optimal uses of cellular immunotherapy sources.
Presenters:
Michael Gustafson, PhD
Mayo Clinic Arizona
USA
Baylor College of Medicine
USA
Fate Therapeutics
USA
MSC-Derived EVs: Cell Therapy without Cells
MAY 26 - 10:00 EDT
Pre-clinical and clinical studies have established that mesenchymal stromal cells (MSCs) yield therapeutically beneficial effects via paracrine action, and that extracellular vesicles (EVs) shed from MSCs contribute to this activity.
However, while efforts are underway to develop MSC-EVs as a standalone therapeutic, many questions remain regarding best practices to achieve a successful outcome.
In this session, key opinion leaders in the field discuss efforts to develop standards to isolate and characterize MSC-EVs (Dr. Sai-Kiang Lim), address critical issues related to how MSC source and particle size influence cargo content (Dr. Bas van Balkom), and evaluate the value of pre-clinical models to assess MSC-EV potency (Dr. Claudia dos Santos).
Therefore, the session is of high value for researchers and clinicians engaged in the study of MSC-EVs and pursuing their use in an experimental clinical therapy.
Presenters:
Don Phinney, PhD
Scripps Research Institute
USA
Bas von Balkom, PhD
University Medical Center Utrecht
Netherlands
Claudia dos Santos, MD
University of Toronto
Canada
Sai-Kiang Lim, PhD
Institute of Molecular & Cell Biology
Singapore
Debating MSC Tissue Sources: Biological and Manufacturing Considerations
MAY 26 - 11:30 EDT
Adipose and bone marrow derived MSCs have met marketing approval in Europe and Japan. The interest in umbilical-cord derived MSCs as an investigational therapy is flourishing and iPS-derived MSCs have emerged as a plausible inexhaustible defined source.
Are there intrinsic biological and manufacturing properties which identify clear front-runner in the off-the-shelf use of MSC and how to evaluate these properties in a clinical perspective?
Presenters:
Karin Tarte, PharmD, PhD
University of Rennes
France
Frank Barry, PhD
National University of Ireland Galway
Ireland
Ivan Martin, PhD
University Hospital Basel
Switzerland
University of Zurich
Switzerland
Kristina Thamm, PhD
denovoMATRIX
Germany
Martha Arango-Rodriguez, PhD
FOSCAL
Colombia
Understanding Regulatory Requirements for Potency Assays
MAY 26 - 15:30 EDT
Marketing approval for MSCs requires the establishment of validated identity and functional attributes for uniform manufacturing as well as to help understand clinical responses in any given indication.
• Will in vitro cell-based assays suffice to meet the bar?
• Is a matrix of functional assays required?
• Do these assays need to correlate with clinical outcomes?
• Can there be consensus amongst international regulators on what these requirements may be for MSC-based therapeutics?
A gap analysis is discussed.
Presenters:
Sowmya Viswanathan, PhD
University Health Network
Canada
U.S. Food and Drug Administration
USA
Mauro Krampera, MD
University of Verona
Italy
Eleuterio Lombardo, PhD
Takeda
Spain
Abstract Presentation
Pallab Pradhan, PhD
Georgia Tech
USA
Abstract Presentation
Razieh Rabani
University Health Network
Canada
Lessons & Recommendations for Clinical Trials of MSCs for Musculoskeletal Diseases in 2021
MAY 26 - 17:00 EDT
This session will explore the state of the art in MSC sourcing and fabrication for disease–specific clinical therapies for musculoskeletal disease. The specific experience of three effective teams in Europe, Japan and Taiwan will be used to catalyze in depth discussion of key challenges for: 1) reproducible product fabrication, characterization and quality control, 2) effective disease-specific targeting, design and execution of rigorous clinical trials for musculoskeletal disease, and 3) both enabling and inhibiting region-specific differences in regulatory environment.
Goals: To provide an authoritative overview of the current status of clinical assessment of culture expanded MSCs in Musculoskeletal Disease.
Methods: Leaders from across the globe are asked to address key strategic and practical issues that have driven and limited current approaches and progress, including:
- Cell Sourcing – Fabrication, Certification
- Disease Setting and Patient Selection
- Delivery Strategies
- Clinical Outcome Assessment
- Evidence to Date from Completed Studies
Presenters:
Christian Jorgensen, MD, PhD
Centre Hospitalier Universitaire de Montpellier
France
Co-Chair
George Muschler MD
Cleveland Clinic
USA
Oscar Lee MD, PhD
China Medical University Hospital
Taiwan
Norimasa Nakamura, MD, PhD
Osaka University
Japan
Felipe Prósper, MD, PhD
Universidad de Navarra
Spain
Allogeneic Therapies – What really is a healthy donor?
MAY 26 - 10:00 EDT
Donor derived cell-based starting materials are fundamental to cell and gene therapies and play a key role in in all aspects from manufacturing to product and patient safety. But what defines a healthy donor and how much characterization is enough?
As allogeneic therapies continue to grow and advance clinically, it will be critical for both therapeutic developers and regulators to harmonize on the requirements and balance safe versus sustainable.
This session will highlight the current state of the industry and debate the outstanding questions impacting harmonized clinical translation and global commercialization.
Questions for consideration:
• What constitutes a healthy donor or healthy enough?
• Starting materials are tested for specified release criteria and with an expectation for these to be defined, but what is realistic?
• What are the characterization requirements (viral testing of the donor to that of the cell-based product for example)?
• There is a need to generate data, but how much is actually enough?
• How do we align to ask the right questions?
Presenters:
Session Chair
Dominic Clarke, PhD
HemaCare, a Charles River Company
USA
Deborah Hursh, PhD
U.S. Food and Drug Administration
USA
David Sourdive, PhD
Cellectis
France
Nkarta Therapeutics
USA
Consulting on Advanced Biologicals Ltd
United Kingdom
Naming Cell & Gene Therapies: Harmonizing with INN Nomenclature and Regulatory Impacts
A Joint Session with the World Health Organization |
MAY 26 - 11:30 EDT
The World Health Organization (WHO) recently published a landmark document in Cytotherapy® on nomenclature in advanced therapy medicinal products (ATMPs). In this breaking session, global leaders representing the different sectors of the CGT field discuss the far-reaching implications of this milestone.
Presenters:
Session Co-Chair
Raffaela Balocco Mattavelli, PhD
World Health Organization
Switzerland
Session Co-Chair
Massimo Dominici, MD
University of Modena and Reggio Emilia
Italy
Ursula Lolzides, PhD
WHO INN Programme
Switzerland
Anthony Ting, PhD
ISCT
Chief Commercialization Officer
USA
Peter Marks, MD, PhD
FDA Center for Biologics Evaluation and Research
USA
Marcos Timón, PhD
AEMPS Unit for Advanced Therapies and Biotechnology
Spain
Tony Manderson, PhD
TGA Cell & Tissue Therapy Unit
Australia
International Alliance of Patients' Organizations
United Kingdom
The Economic Impact on Healthcare and the Future of Advanced Cell & Gene Therapy
MAY 26 - 15:30 EDT
Despite the unprecedented difficulties of the pandemic, it is still an exciting time for Cell and Gene Therapy (CGT). Currently there is over one thousand ongoing clinical trials in the field. There is a buzz about the potential for advanced CGT products to decrease healthcare costs and deliver patient value, but cost and patient access are still significant issues that may require innovative funding models. Strong clinical results are attractive for investors in CGT companies, but limited examples of commercial success thus far may still present a barrier to accelerating investment.
The session will cover:
• The state of cell and gene therapy, focusing on investment and financing
• Strategic business development for the commercial model of CGT including clinical manufacturing and product design
• Development of strategies for reimbursement and how to define the value of these innovative therapies
Presenters:
Julie Allickson, PhD
Wake Forest Institute of Regenerative Medicine
USA
Patrick Rivers, MBA
Aquilo Capital
USA
Reni Benjamin, PhD
JMP Securities LLC
USA
Matthew Durdy
Cell and Gene Therapy Catapult
UK
Stephanie Farnia MPH
Nimitt Consulting
USA
Advancing the translational process through automation & equipment that deliver successful scale up/out in cell therapies
MAY 26 - 17:00 EDT
Successful translation of cell based therapy relies heavily on a combination of research, process development, and manufacturing. What have the successes of early commercial CAR-T products and late stage cell therapies taught us? What hurdles did the industry have to overcome, and how will new technologies impact the translational process? How has the definition of “automation” changed, and what does industry need?
Presenters:
John McMannis
Industry Consultant
USA
OriBiotech
UK
Cellares
USA
PACT Pharma
USA
University of Pennsylvania Perelman School of Medicine
USA
Drug Delivery: The Therapeutic Power of Engineered EVs
MAY 27 - 10:00 EDT
EVs are part of an ancient intercellular communication system we recently have become aware of. They transmit complex signals in physiological and pathophysiological situations, in a targeted manner. Thus, EVs provide the ideal shuttle for drug delivery. To this end it is a challenge in the field to unravel the EVs tropism and to develop strategies for EV loading. In this session, sophisticated technologies are introduced for the genetic engineering and targeting of EVs as shuttle systems for biotherapeutics.
Presenters:
Bernd Giebel, PhD
University of Duisburg-Essen
Germany
Chulhee Choi, MD, PhD
KAIST Institute
South Korea
Samir El Andaloussi, PhD
Karolinska Institute
Sweden
Le Thi Nguyet Minh, PhD
National University of Singapore
Singapore
Jiajun Shi
National University of Singapore
Singapore
David Bernal, PhD
University of Murcia
Spain
Extracellular Vesicles as Future Therapeutics and Indicators of Cardiac Disease
MAY 27 - 11:30 EDT
Extracellular vesicles (EV) are a potent component of the secretome, and as such have been harnessed recently as therapeutics and biomarkers of cardiovascular disease. The objective of this session is for attendees to appreciate the wide biological and therapeutic potential for EV to improve our understanding and management of cardiovascular disease, in both humans and naturally occurring companion animal disease models.
Presenters:
Andrew Hoffman, DVM, DVSc
University of Pennsylvania
USA
Dominique de Kleijn, PhD
UMC Utrecht
Netherlands
Saumya Das, MD, PhD
Massachusetts General Hospital
USA
Eduardo Marban, MD, PhD
Cedars-Sinai
USA
Vicky Yang, DVM, PhD, DACVIM
(cardiology)
Tufts University
USA
Federica Marinaro, MSc
Centro de Cirujia de Minima Invasion Jesus Uson, Spain
Marta Monguió-Tortajada, PhD
Germans Trias i Pujol Research Institute, Spain
EVs for Infectious Diseases and Preparedness for Future Pandemics
A Joint Session with: | ![]() ![]() |
MAY 27 - 17:00 EDT
Description Forthcoming
Presenters:
Sai-Kiang Lim, PhD
Institute of Molecular & Cell Biology
Singapore
Daniel Weiss, MD, PhD
University of Vermont
United States
Lorena Martin-Jaular, PhD
Institut Curie
France
Yong Song Gho, PhD
Pohang University of Science and Technology
South Korea
Michael Bellio, PhD
Organicell Regenerative Medicine Inc.
USA
Traceability and Labeling for Cell-based Therapies and Regenerated Tissues
MAY 27 - 15:30 EDT
Effective traceability, unique identification and standardized labeling are all important ways to enhance safety of clinical product during manufacturing, release and distribution to the patient. The need for traceability is well recognized but the complexity of the traceability pathway for medical products of human origin (MPHO) is poorly understood. ISBT 128 is the global standard for the terminology, identification, coding and labeling of MPHO, which includes cell-based products, regenerated tissues and many other products of human origin (including blood, cell, tissue, milk, and organ products). It provides international consistency to support the transfer, traceability and transfusion/transplantation of these products. The session will provide an overview of traceability for MPHO, and highlight recent developments of ISBT 128 to support standardized labeling of collection products for further manufacture, and the availability of product description codes to support nonproprietary names and novel products in clinical trials. In addition to the presentations, a round-table forum will provide an opportunity for delegates to pose questions to the panel.
Presenters:
Lizette Caballero, BSc, MLS(ASCP)CM
The Janssen Pharmaceutical Companies of Johnson & Johnson
USA
Paul Ashford, MSc
ICCBBA
United Kingdom
Martin Hildebrandt, MD
ICCBBA
Germany
Julie Allickson, PhD
Wake Forest Institute of Regenerative Medicine
USA
Dilemmas in Transplantation for Acute Myeloid Leukemia
MAY 27 - 10:00 EDT
We live in an exciting time for the treatment of acute myeloid leukaemia. After decades of frustration, recent progress in the understanding of the pathogenesis of AML, coupled with novel therapeutic innovations, are rapidly changing the options available to patients. While allogeneic transplantation remains central to the treatment of high-risk AML, clinicians are now faced with difficult decisions when trying to optimise care for such patients. How should chemotherapy be used to optimise disease status prior to transplant? Which alternative donor should be chosen for patients with high-risk AML lacking a matched donor? Are maintenance strategies or novel cell therapies relevant post-transplant? These dilemmas will be discussed by five exciting speakers in this clinically oriented session, which will include a debate and ample room for audience participation.
Presenters:
Duncan Purtill, MD
Fiona Stanley Hospital
Australia
Robert Wynn, MD, MB BChir, MRCP FRCPath
Central Manchester University Hospitals NHS Foundation Trust
UK
Juliet Barker, MBBS
Memorial Sloan Kettering Cancer Center
USA
Charles Craddock, CBE, FRCP (UK), FRCPath, DPhil
University of Birmingham
UK
David Curtis, MBBS, PhD, FRACP, FRCPA
Monash University
Australia
Kanya Rajangam, MD, PhD
Nkarta Therapeutics, Inc.
USA
Debating PTCy vs T-Cell Depletion for Treating Non-Malignant Diseases: Do we know which one is better?
MAY 27 - 11:30 EDT
Hematopoietic stem cell transplantation is curative for many nonmalignant disorders, including primary immune deficiency disorders (PIDD), primary immune regulatory disorders (PIRD), hemoglobinopathies, inherited bone marrow failure syndromes (IBMFS), and metabolic disorders. Historically, success has been limited by the scarcity of fully matched donors. More recently, the use of alternative donors, including mismatched family members, unrelated bone marrow and peripheral blood, or unrelated cord blood, has dramatically expanded the donor’s availability. During this session experts in the field will debate about the use of different stem cell sources and will discuss alternative strategies of in vivo and ex vivo graft manipulation strategies with a special focus on the use of PT-Cy versus alpha/beta T-cell depletion.
Presenters:
Alice Bertaina, MD, PhD
Stanford Medical School
USA
Carmem Bonfim, MD, PhD
Federal University of Paraná
Brazil
Amy DeZern, MD, MHS
John Hopkins Medicine
USA
Orly Klein, MD
Lucile Packard Children's Hospital Stanford
USA
Kanchan Rao, MD
Great Ormond Street Hospital
UK
Parinda Mehta, MD
Cincinnati Children's Hospital
USA
Joseph Oved, MD, MA
Children's Hospital of Philadelphia
USA
State of the Art in Cord Blood Transplantation: Banking, Expansion and Future Horizons - ISCT-WMDA Joint Session
A Joint Session with: | ![]() ![]() |
MAY 27 - 15:30 EDT
Cord Blood (CB) has unique properties and advantages, including a strong anti-tumor effect. This session will focus on how the use of cryopreserved cord blood cells is changing from the standard transplant to engineered stem cells and T cells directed to viral or disease specific targets. The future of cord blood banking and the new strategies to overcome the main limitations of the stem cell content (including the results of phase II and phase III clinical trials) will be discussed to provide a broad overview of new cord blood applications
Presenters:
Allistair Abraham, MD
Children's National Hospital
USA
Annalisa Ruggeri, MD, PhD
San Raffaele Hospital
Italy
Catherine Bollard, MD, MBchB
Children's National Hospital
USA
Sandra Cohen, MD
Université de Montréal
Canada
May Daher, MD
MD Anderson Cancer Center
USA
Mitchell Horwitz, MD
Duke University
USA
Sergio Querol, MD, PhD
Banc de Sang i Teixits
Spain
Andromachi Scaradavou, MD
Memorial Sloan Kettering Cancer Center
USA
Tuning the Immune System to Optimize the Efficacy of Cellular Therapies
MAY 27 - 17:00 EDT
The talks in this session will highlight different components of effective immune reconstitution after hematopoietic stem cell transplant. Thymic dependent and thymic independent pathways for T cell reconstitution as well as novel validated milestones of T cell immune reconstitution will be discussed. The important contribution of the innate immune system to protection from infection and relapse after transplant will be discussed in the second segment.
Presenters:
Susan Prockop, MD
Memorial Sloan Kettering Cancer Center
USA
Stanford Children's Health
USA
Memorial Sloan Kettering Cancer Center
USA
UMC Utrecht
Netherlands
MD Anderson Cancer Center
USA
New Horizons for Cell Therapies to Treat Cardiovascular Diseases
MAY 28 - 10:00 EDT
A few years ago, great expectations were set around the clinical potential of cell therapy for the treatment of ischemic heart disease. Initial excitement, derived from encouraging preclinical data, resulted in a rapid translation into clinical research. The puzzling outcome of the first clinical trials mitigated the initial enthusiasm both among cardiologists and the various stakeholders. However, some more recent clinical studies suggest that perhaps, especially in the most severe patients, the correct use of reparative strategies could be of help. These latest results will be presented, and possible future strategies discussed during this session.
Presenters:
Massimiliano Gnecchi, MD, PhD
University of Pavia
Italy
Hartcentrum OLV Aalst
Belgium
Leuven University
Netherlands
Centro Cardiologico Monzino IRCCS
Italy
Networks and Dynamics in the Clinical Translation of Safe and Effective Cellular Therapies for Musculoskeletal Diseases
MAY 28 - 11:30 EDT
This session takes a broad perspective on the translation of safe and effective cellular therapies for musculoskeletal disease. The presentations and following forum will explore the current dynamics between biomedical innovation, clinical literature, clinical research networks, professional societies, physician practices, and patient education.
Dr. Guilak, a leading bioengineer and past president of the Orthopaedic Research Society, will define the state of clinical translation in the key area of cartilage tissue replacement.
Dr. Piuzzi, an orthopaedic surgeon and clinical-scientist, will define the current evidence for the clinical efficacy (derived from systematic reviews and RCTs) of cellular injection therapies for osteoarthritis, osteonecrosis, tendonopathy and fracture non-union.
Dr. Maloney, past president of the American Academy of Orthopaedic Surgery and current Chair of the AAOS Clinical Registry Program will illuminate the investments being made to develop digital reporting tools and widespread professional adoption of clinical registries as a resource for rigorous longitudinal assessment and comparison of implants, surgical techniques, as well as cellular “biologic” therapies that are currently regulated by the FDA under 361 exemptions as HCT/Ps.
Presenters:
George Muschler, MD
Cleveland Clinic
USA
William J Maloney, MD
Stanford University
USA
Farshid Guilak, PhD
Washington University School of Medicine
USA
Nicolas Piuzzi, MD
Cleveland Clinic
USA
Jesil Kasamkatill, MSc
University Hospital Basel
Switzerland
Judith Krieger, MSc
Fraunhofer Institute
Germany
Regenerative Engineering for Nervous System Diseases
MAY 28 - 15:30 EDT
This session will explore the state of the art in sourcing and fabrication of neurons for disease–specific clinical therapies. The specific experience of four highly productive teams will be used to catalyze in depth discussion of key challenges for 1) reproducible cell product fabrication, characterization and quality control, 2) effective disease-specific targeting and cell delivery strategies, 3) design and execution of rigorous clinical trials, and 4) key technical challenges or gaps that must be addressed to enable more effective development and translation of cell therapy for neurological tissue regeneration.
Presenters:
Budd Tucker, PhD
University of Iowa Health Care
USA
Molly Shoichet, FRS
University of Toronto
Canada
Clive Svendsen
Cedars-Sinai Medical Center
USA
Cell and Biomaterials based Technologies in GI Bioengineering: Approaching the Bedside
MAY 28 - 17:00 EDT
The session features three speakers. Paolo De Coppi will speak about the application of extracellular matrix-based scaffolds as a platform for the bioengineering of the pediatric esophagus. Ekaterine Berishvili will illustrate how a combinatorial approach harnessing stem cell engineering, gene editing and placenta extracellular matrix-based biomaterials may foster progress in beta cell replacement for type 1 diabetes. Geraldine Mitchell will elucidate on progress in the bioengineering of the vascular network of complex tissues. Overall, this session will offer an overview of the state of the art of tissue engineering technologies as they may be applied to GI medicine.
Presenters:
Rachele Ciccocioppo, MD
University of Verona
Italy
Co-Chair
Vincenzo Cardinale, MD, PhD
Sapienza University of Rome
Italy
Paolo De Coppi
Great Ormond Street Hospital for Children
UK
Ekaterine Berishvili, MD, PhD
University of Geneva
Switzerland
Geraldine Mitchell, MBChB
St. Vincent's Hospital Melbourne
Australia
Pradyut Paul, PhD
University of Wisconsin-Madison
USA
Lymphodepletion and Biomarkers in Cell Therapy
MAY 28 - 10:00 EDT
Description Forthcoming
Presenters:
Jaap Jan Boelens, MD, PhD
Memorial Sloan Kettering
USA
Co-Chair
Michael Scordo, MD
Memorial Sloan Kettering
USA
Children's Hospital of Philadelphia
USA
Fred Hutchinson Cancer Research Center
USA
Vanessa Fabrizio, MD, MS
Duke University
USA
Novel Allo-Engineering Approaches and Progress in Off-the-Shelf Products
MAY 28 - 11:30 EDT
This session will discuss various approaches to allogeneic cell products, including off-the-shelf virus-specific T cells, allogeneic CAR T cells, and allogeneic gamma delta T cells. What makes this session unique is that parts of it will focus on genetic approaches to evading immune rejection and increasing persistence while others will present on non-gene therapy-based approaches to allogeneic therapies and why they are an appealing alternative to autologous cell and gene therapies.
Presenters:
Patrick Hanley, PhD
Children’s National Hospital
USA
Co-Chair
Margaret Lamb, MD
Children’s National Hospital
USA
Maksim Mamonkin, PhD
Baylor College of Medicine
USA
Jason Dubovsky, PhD
Atara Biotherapeutics
USA
Lawrence Lamb, PhD
IN8Bio, Inc.
USA
Michelle Cox, M.S.
Mayo Clinic
USA
Cristina Ferreras, PhD
IdiPAZ
Spain
Progress in In-Vivo Gene Therapies
MAY 28 - 15:30 EDT
To help attendees learn and understand fundamentals of AAV virology as it applies to in vivo gene therapy applications. We will also cover the current state of the art AAV technologies being applied preclinically as well as clinically in both the CNS and blood disorders.
Following the seminar, the participant will be able to:
• Explain and understand the current state of field of AAV gene therapy.
• Discuss the potential for AAV gene therapies to be applied to both brain and blood disorders.
Presenters:
Sandeep Soni, MD
CRISPR Therapeutics
USA
Co-Chair & Speaker
Nicole Paulk, PhD
University of California San Francisco
USA
Krystof Bankiewicz, MD, PhD
Ohio State University
USA
Lindsey A. George, MD
University of Pennsylvania
USA
Cell Therapy and the Spleen: Modulating Therapeutic Outcomes After Inflammatory Crises or Injury Onset
MAY 28 - 17:00 EDT
Regardless of organ system (CNS, Cardiopulmonary, Severe body trauma), after onset of severe injury to these systems, the peripheral immune system is engaged and, in most cases, results in a secondary “pathophysiological hit” subsequent to the initial injury, resulting in enhanced secondary damage at the site of initial injury.
The engagement of the peripheral immune system in the acute time frame after initial injury onset, results in increased activation and infiltration of leukocytes, myeloid cells, neutrophils and increased levels of inflammatory cytokines.
Presenters:
Robert Mays, MD
Athersys, Inc.
USA
Co-Chair
Charles S. Cox, MD
University of Texas Medical School
USA
Sangeeta S. Chavan, PhD
Feinstein Institutes for Medical Research
USA
Richard Maziarz
Oregon Health & Science University
USA
Sean Savitz
University of Texas Medical School
USA
Translating Cellular Therapies from Bench to Clinic – The Critical Role of GMP-compliant, Scalable Cell Engineering Technology
MAY 26 - 10:00 EDT
Increasingly sophisticated approaches to cellular engineering, including CRISPR-mediated gene editing and expression of disease-targeting receptors, require corresponding advances in cellular delivery technologies.
Aside from the high manufacturing costs of viral vectors, demand for non-viral methods is driven by the diversity of cell types (e.g,, T cells, NK cells, stem cells) and loading agents (e.g., RNPs, mRNA, transposons) that are central to many engineered cellular therapies. Moreover, aggressive development timelines combined with strict standards for safety and therapeutic potency create a need for cell modification protocols that translate seamlessly from the research lab into the clinic, accommodating a broad range of processing scales without compromising cell viability or loading efficiency.
This Industry Symposium brings together leading researchers and clinicians to discuss their experiences with translating and scaling non-viral approaches to engineered cellular therapies.
Presenters:
James Brady, PhD, MBA
MaxCyte
USA
Children Hospital of Los Angeles (CHLA)
Keck School of Medicine, USC
USA
City of Hope, Laboratory for Cellular Medicine
USA
University of California, San Francisco
USA
Washington University School of Medicine
USA
Defining Intelligent Scale Up and Scale Out. How Small Decisions Have Major Implications for Intelligent Scale Up And Out
MAY 26 - 15:30 EDT
Join a stellar panel of industry leaders as they speak about how you can define and plan for intelligent scaling of cell and gene therapies. Drawing on insights from academic, industry, facility design, and tool providers, this panel will explore a variety of decision points researchers and drug developers face when scaling up and scaling out. Please participate in the live polling and feel free to contribute questions during the Q&A session at the end!
Presenters:
Juan Vera, MD
Marker Therapeutics
USA
Precision Biosciences
USA
AmplifyBio
USA
PACT Pharma
USA
ElevateBio, BaseCamp
USA
Autolus Therapeutics
USA
Kicking Off Cell Therapies With the Right Materials – Experts Weigh In
MAY 27 - 10:00 EDT
Cytiva has gathered a panel of experts to weigh in on the standardization of cell therapy starting materials. With backgrounds ranging from the clinic to commercial, these cell therapy leaders will share insight, experience, and perspective related to the following:
• Case studies from both start-up and established companies preparing to manufacture therapeutic products for Phase 1 and Phase 2 trials
• Establishing best-in-class cryopreservation programs
• The importance and challenges of ensuring standardization of cell therapy materials, from a global perspective
Presenters:
Gary M. Pigeau, PhD
Cytiva
USA
AllCells, LLC
USA
CCRM
Canada
Scottish National Blood Transfusion Service
Canada
Expanding the Possibilities of Cell Therapy
MAY 27 - 11:30 EDT
Join Miltenyi and leading cell therapy experts for case study presentations and a panel discussion featuring live Q&A on expanding the possibilities of cell therapy.
Boris Fehse, PhD will share his work regarding the establishment of a mRNA electroporation protocol for the automated production of CCR5-edited CD4+ T cells on the CliniMACS Prodidy® to prevent HIV infection.
Sandy Pelletier, PhD will outline her approach to isolate tumor-reactive T cells from solid tumors using the MACSQuant® Tyto® Cell Sorter.
Moonsoo M. Jin, PhD will discuss his work with affinity-tuned CAR T cells to target solid tumors with high-level antigen expression.
Session Chair
Miltenyi
Germany
University Hospital Hamburg
Eppendorf
Germany
CHUM Research Center
Canada
Molecular Imaging Innovations Institute (MI3)
USA
Current and future state of CAR-T cell therapy manufacturing- Closing, automating and preparing for the allogeneic revolution
MAY 27 - 15:30 EDT
The commercial launch of approved cell therapies demonstrated to the industry and world alike the transformative impact they can have on a patient’s life and revolutionize the treatment of human disease. As a result, it is critical to evaluate and identify what is needed by the industry to support both the manufacture of small-scale autologous CAR-T cell therapies and adapt manufacturing processes towards large-scale allogeneic therapies that can impact a broader patient population. In this 90 minute symposium session, we will hear updates from industry leaders on the current status of their research in the CAR-T manufacturing space and gain an understanding of the challenges associated with supporting conversion of autologous to allogeneic workflows. To present a complete overview, the panelists listed below will focus on the following:
• Closed modular cell therapy workflow solutions
• Harnessing next-generation genome editing to develop allogeneic cell therapies
• Improving therapeutic success through process development and allogeneic manufacturing
Presenters:
Xavier De Mollerat Du Jeu, PhD
Thermo Fisher Scientific
USA
Thermo Fisher Scientific
USA
Thermo Fisher Scientific
USA
Genentech
USA
Caribou Biosciences
USA
Cellicon Valley: Building A Thriving Cell and Gene Therapy Ecosystem Leveraging Academia and Industry in Greater Philadelphia
MAY 27 - 17:00 EDT
Since the founding of America’s first hospital, Pennsylvania Hospital, by Benjamin Franklin and Dr. Thomas Bond in 1751, Philadelphia has had a longstanding commitment to healthcare innovation.
Today, that legacy lives on in Philadelphia and is exemplified by the emergence of numerous paradigm shifting and life-changing medicines including the first gene therapy approved by the U.S. Food and Drug Administration (FDA), Kymriah™, as well as the first directly administered gene therapy approved in the U.S. that targeted a disease caused by mutations in a specific gene, LUXTURNA™. Both therapies are based on fundamental technology breakthroughs from the University of Pennsylvania and the Children’s Hospital of Philadelphia (CHOP), and both were developed in close partnership with industrial collaborators helping to drive the emergence of “Cellicon Valley” in Philadelphia over the last decade.
More than just a name, “Cellicon Valley” is home to world-class academic research institutions that bring in over $1B in NIH funded research dollars per year, more than 35 leading cell and gene therapy companies that have collectively raised collectively raised over $1.5B in capital in 2020, and over 1.5 million square feet of new lab space currently in development. This burgeoning ecosystem is a unique environment that truly leverages academia and industry to foster the needed skills and experience to further innovate, test, and commercialize new therapies.
Join us to learn more about the origins, models for collaboration, and future direction of “Cellicon Valley”.
Presenters:
John S. Swartley, MBA, PhD
UPenn, Penn Center for Innovation
USA
Carisma Therapeutics
USA
Cabaletta Bio
USA
Tmunity
USA
The Development Trend and Clinical Application of Novel Automated CAR T-cell Expansion Technology
MAY 28 - 10:00 EDT
CAR T cells are the trending immunotherapy technology for the treatment and cure of malignant tumors. They are a "living medicine," and the preparation of high-quality and reliable CAR T cells (products) is one of the key factors in CAR T-cell drug formation. The clinical efficacy and safety of CAR T cells depend not only on the design of the chimeric antigen receptor (CAR) gene, but also on the composition, quantity, status, and function of T immune cells. Traditional CAR T-cell preparation technology has been unable to meet the needs of clinical treatment. Here, we describe a modern CAR T-cell expansion technology using an automated platform, the Quantum® Cell Expansion System (Terumo Blood and Cell Technologies). We shifted our existing manual process for T-cell sorting, activation, and transduction to Quantum-based automated expansion. We observed that obtaining a clinical dose (2 × 109 to 3 × 109 CAR T cells) for hematological malignancies in an investigator-initiated trial (IIT) took only 7 days on Quantum, compared to up to 13 days using manual techniques. This shortened expansion time may help to reduce turnaround time.
With continuous improvement and breakthroughs in the technology of CAR T-cell production, CAR T cells can really improve anti-tumor efficacy, reduce tumor recurrence, and improve safety.
Presenters:
Augustus Kilgore, PhD
Terumo BCT
USA
Shanghai Unicar-Therapy Biomed-Pharmaceutical Technology Co.,Ltd,
China
Strategies for Implementing Rapid Mycoplasma Testing Methods to Advance QC in Cell and Gene Therapy Manufacturing
MAY 28 - 11:30 EDT
Cell and gene therapies are unique, warranting a new look at analytical development and QC management in order to establish relevant and sustainable strategies to produce safe, yet commercially viable products that meet regulatory requirements. To create state-of-the-art manufacturing efficiency, rapid microbiological methods (RMMs), or alternative microbiological methods, are essential because traditional culture-based methods do not provide same-day assay turnaround time, lower sample volume utilization, or often a way to automate or scale testing, limiting their suitability and in-house use. There are ways to incorporate RMMs for QC testing, such as using a nucleic acid amplification technique as an alternative mycoplasma testing system after appropriate product-specific validation. Yet, uncertainty around supplier selection and technical and regulatory requirements impedes cell and gene therapy manufacturers from adopting RMMs for routine QC testing. During this session, Roche CustomBiotech brings industry experts to share their in-depth case studies on selecting and adopting a commercial rapid PCR-based mycoplasma kit in hopes to help push innovative RMMs forward in an industry that needs them.
Presenters:
Evonne R. Fearnot, MSBME, MBA
Roche CustomBiotech
USA
Celyad Oncology
Belgium
University of Toronto
Canada
Successes and Failures Managing Clinical Trials In Apheresis Centers
MAY 26 - 12:30 EDT
Sponsored by:
How can apheresis centers plan for the growing demand of clinical and commercial cell and gene therapy products?
This roundtable discussion will focus on key lessons learned from running cell and gene therapy clinical trials in apheresis centers. Hear panelists discuss critical issues including: data integrity and centralization, opportunities for standardization in approaches, tools, as well as systems that support operational efficiency and minimize risk, which ultimately support health care professionals and their patients in apheresis centers.
Panelists:
Dr. Joseph (Yossi) Schwartz
Weill Cornell Medicine
USA
UC Health
USA
Vineti
USA
Optimizing Vein to Vein – Challenges with donor characterization, starting material collection & distribution
MAY 27 - 12:30 EDT
Sponsored by:
What are common pain points in the vein to vein pathway?
This roundtable discussion will focus on how companies can execute and perfect a seamless chain of custody to ensure the quality and integrity of cell and gene therapy products from donor to patient (vein to vein). Hear from panelists about navigating relationships with key stakeholders including hospital partners, and considerations developers should keep in mind as we collaborate to build a sustainable ecosystem that can supports the manufacturing and scale-up of an increasing number of cell and gene therapy products.
Panelists:
Mark Lowdell, PhD, FRCPath, FRSB
UCL
UK
Be The Match BioTherapies
USA
Cytiva
USA
CSO Cryoport Inc.
USA
Lonza
USA
Preparing for the Future of CAR T Manufacturing
MAY 28 - 12:30 EDT
Sponsored by:
What are critical early stage questions developers should be asking to prepare for future manufacturing success?
This roundtable discussion will focus on the hurdles facing CAR T manufacturing including the strategies, tools, and technologies that can be used to minimize the pain points in development. As the autologous vs allogeneic debate continues, hear the roundtable’s experts discuss delivery technologies and key partners who should be engaged to reduce COGs and optimize process development & manufacturing of CAR T therapies.
Panelists:
Bruce Levine, PhD
University of Pennsylvania
USA
Avectas
USA
Berkeley Lights, Inc.
USA
David DiGiusto
Resilience
USA
Accelerating Clinical Readiness and Speed to Market of CAR T Therapies
MAY 28 - 15:00 EDT
Sponsored by:
What platforms and services exist to save time and cost in the development of cell and gene therapies?
Expert panelists in this roundtable will discuss process development and focus on buy vs build facility considerations that should be evaluated early in development to set up for future success. The roundtable will also touch on how to balance the race between advancing to the clinic, with the need for a robust manufacturing process that limits future comparability studies.
Panelists:
Patrick Hanley, PhD
Children’s National Medical Center
USA
Miltenyi Biotec
USA
G-CON Manufacturing
USA
Lykan Biosciences
USA
Implementation of Advanced QC Testing Solutions for Cell and Gene Therapy Manufacturing
Available On-Demand on May 26
During this tutorial, industry experts will highlight needs and challenges for advancing QC testing for C> manufacturing. A first presentation will be dedicated to microbial contamination control and testing strategies for just in time release of CAR T products. A second presentation will focus on novel strategies for phenotypic cell analysis in cell therapy and implementation of the Accellix platform.
Presenters:
Félix A. Montero Julian, PhD
bioMerieux
France
Rey Mali
Accellix
France
Bristol Myers Squibb
USA
Bristol Myers Squibb
USA
Challenges in Scaling Analytics for Cell & Gene Therapies
Available On-Demand on May 26
During this course several industry leaders will present some of the challenges associated with scaling process analytics for the commercial manufacture of Cell and Gene therapies, as well as innovative strategies and technologies for overcoming these challenges.
Learning Objectives:
• How to anticipate challenges when scaling up and scaling out process analytics
• How to mitigate risk when scaling your analytics
• How automation can simplify your go to market strategy
• How automation leads to more standardized results
Presenters:
Session Chair
Colin Merrifield
Bio-Techne
USA
Chris Heger, PhD
Bio-Techne
USA
Kiren Baines, PhD
eXmoor Pharma Concepts
UK
A Case Study: Streamlining Digital Transformation and Reducing Data and Process Silos in Biologics
Available On-Demand on May 26
Biologics manufacturing and new classes of precision therapeutics, including cell and gene therapies, require a new category of companion informatics that automates and digitalizes complex manufacturing, quality management, and clin-ops processes. This digital transformation can reduce time to market, reduce development costs, and streamline the scaling and development of these medicines. This discussion will feature an in-depth discussion on digital transformation for advanced therapeutics including biologics manufacturing, and a case study with a leading CDMO company.
Why this is an important topic:
As the need to digitalize increases to improve productivity and technology innovations for biologics manufacturers, companies like L7 Informatics and Cognate BioServices are taking the lead in collaborating to unify systems across research, manufacturing, and clinical operations.
Presenters:
John Conway
20/15 Visioneers
USA
L7 Informatics, Inc.
USA
Cognate BioServices
USA
Pall's Approach to Viral Vector Manufacturing Addressing Your Biggest Challenges
Available On-Demand on May 26
Pall’s Process Development Scientists have worked with dozens of clients on an array of vaccines, gene and cell therapies, many already marketed, literally from A (AstraZeneca Covid 19 vaccine) to Z (Zolgensma). Leverage their expertise.
Viewers will learn how to:
• decide between suspension or adherent cell culture?
• separate empty from full capsids in viral vector manufacturing?
• develop an industrial scale Lentivirus production platform?
Presenters:
Jennifer Moody
Pall Corporation
Canada
Pall Corporation Research and Development
USA
Pall Corporation Research and Development
USA
Pall Corporation
USA
Key Considerations in Scalable Regenerative Medicine Manufacturing Process Development and Next Generation MSC3.0 Product Formats
Available On-Demand on May 26
Developing allogeneic cell therapy products with scalable manufacturing can take years of time and costs millions of dollars.
This presentation will highlight development pain points and address:
• Key strategic decisions during product development that drive manufacturing platform decision making
• Raw material strategies that reduce product & process development timelines and cost while streamlining the path towards hMSC-based clinical manufacturing
• Key considerations for implementing standardized large scale bioreactor processes for hMSC manufacturing while maintaining comparability in quality parameters
• Complete solutions for next generation hMSC-based extracellular vesicle production and genetically engineered hMSC products
Presenters:
RoosterBio Inc.
USA
RoosterBio Inc.
USA
Scalable Xeno-free Production of MSC-Derived EVs in Single-use Bioreactors
Available On-Demand on May 26
Learn how EVs are robustly produced from various hMSC (BM, AT & UCM) using a scalable serum-/xeno-free microcarrier-based culture platform, employing a single-use bioreactor and human platelet lysate culture supplement (UltraGROTM-PURE).
Achieving production of clinical EV doses will require cost-efficient, scalable, GMP ex-vivo expansion systems. Here we present a readily scalable, xeno-free MSC-EV expansion approach in a microcarrier-based bioreactor using human platelet lysate supplement;
• Producing MSC-derived EVs from various human tissue sources; bone marrow (BM), adipose tissue (AT), and umbilical cord matrix (UCM)
• Experience in a Vertical WheelTM Bioreactor
• Results using xeno-free UltraGROTM-PURE (FDhPL) supplement
Presenters:
Claudia Lobato de Silva, PhD
University of Lisboa
Portugal
Miguel de Almeida Fuzeta, PhD Candidate
iBB, IST, U. Lisboa
Portugal
Identify and Select Optimal T Cell Phenotypes
Available On-Demand on May 26
Learning Objectives:
Understanding the underlying mechanisms of T cell differentiation and function is critical for developing effective therapeutics. However, T cells are extremely heterogeneous and have variable responses to stimulation. This makes predicting specific Tcell responses extremely difficult. To identify effective T cells within a heterogeneous sample, scientists must be able to characterize T cells at the single-cell level. However, most existing single-cell methods are destructive and prevent the correlation of cytokine secretion with other functional parameters like cytotoxicity or gene expression. Here we share use cases that demonstrate how the Opto™ Cell Therapy Development workflow makes it possible to fully characterize individual T cells, correlate key characteristics, and directly link phenotype to gene expression at a single-cell level—all on the same T cells—enabling the development of more efficacious therapies.
Presenter:
Berkeley Lights, Inc.
USA
BioCentriq Cell & Gene Therapy Development & Manufacturing Center
Available On-Demand on May 26
Learning Objectives:
•Unique technology agnostic CDMO with “hoteling” approach
•Open and willing to adopt innovative approaches and technologies
•Additional focus on workforce development
Presenter:
SVP and General Manager
BioCentriq
USA
T & NK Cell Culture: Novel Substitute for Human AB Serum
Available On-Demand on May 26
BioLegend is a global trusted partner of premier research institutions, biotechnology, and pharmaceutical companies. Our cutting-edge tools provide reproducible results in immunology, oncology, neuroscience, stem and proteogenomics.
During this presentation, learn:
• To show how serum replacements can benefit the immunotherapy field.
• About our new product, Cell-Vive™ T-NK Xeno-Free Serum Substitute, GMP. Designed to work in combination with multiple basal media, Cell-Vive™ performs similarly or better than human AB serum, for both T and NK Cells.
Presenter:
Scientist III
BioLegend
USA
Everything is rethinkable - robust and scalable manufacture of human cells via cellular reprogramming
Available On-Demand on May 26
Learning Objectives:
• Introduction to novel technology that allows consistent, fast and scalable manufacturing of human cells from hiPSCs.
• Understanding the role of transcription factors in cell reprogramming.
• How this applies to cell therapy.
Presenter
CEO & Founder
bit.bio
UK
Manufacturing Considerations for MSCs and Broader Allogeneic Modalities
Available On-Demand on May 26
Learning Objectives:
• Understand challenges associated with scalability of MSCs
• Catalent’s solutions to accelerate commercial-scale manufacturing of MSC and other allogeneic therapies
Presenter
Catalent Cell and Gene Therapy
USA
What’s Next for AAV-based Gene Therapy?
Available On-Demand on May 26
Learn about:
• Overview of AAV-based gene therapy, including challenges and potential solutions.
• Therapeutic Expansion – Learn how to conduct a systematic database-driven selection of new indications in the gene therapy space
Presenter:
Principal
Catenion
Germany
De-Risking Your CAR-T Manufacturing Process and Improving Efficiency
Available On-Demand on May 26
Viewers will understand how to de-risk your CAR-T process by:
• developing closed systems
• utilizing automated processes
• establishing modular manufacturing approaches & optimizing each unit operation
Presenter:
CCRM
Canada
Manufacturing Cell and Gene Therapy – Commercial Supply vs Hospital Supply
Available On-Demand on May 26
In identifying a manufacturing partner for your cell-based therapy, several options exist for early phase clinical translation. A major decision point is whether to work with a commercial CDMO or an academic/hospital-based supplier. Which of these will enable better clinical integration and patient care? How will these suppliers scale with your program over time? Associate Professor Dominic Wall, Interim CEO and Chief Scientific Officer at Cell Therapies Pty Ltd and Executive Director of Business Ventures at the Peter MacCallum Cancer Centre, will provide his perspectives on these challenging questions.
Cell Therapies Pty Ltd (CTPL) is one of the most experienced contract manufacturers of global cellular therapy products. Since 2003, CTPL has provided GMP manufacturing services to meet international regulatory standards for clients. We operate a 10-cleanroom facility in Melbourne that holds commercial and clinical trial manufacturing licenses by Australia’s TGA.
Learning Objectives of this session is understanding the advantages and challenges in working with an academic or hospital manufacturer vs a commercial CDMO, especially with respect to:
- Clinical integration
- Patient care
- Scalability and infrastructure
- Cost drivers – budget vs growth
Presenter:
Cell Therapies Pty Ltd
Peter MacCallum Cancer Centre
Australia
Automated Cell Manufacturing for Rigor and Reproducibility in Process Development and Production
Available On-Demand on May 26
Presenter:
Cleveland Clinic
USA
Introducing the Most Advanced Ultra Cold Shipper, Cryoport Elite™
Available On-Demand on May 26
The Cryoport Elite™ multi-use shipper is aimed to sustain consistent temperatures during transit for longer than 4 days, prevents accidental openings during shock events, and provides real-time temperature monitoring on the outside of the shipper.
Presenter:
Vice President, Business Development, Americas
Cryoport Systems
USA
The Cymerus™ iPSC-derived MSC Platform
Available On-Demand on May 26
Cymerus™ is a clinical stage, iPSC-derived therapeutic stem cell platform technology.
Learning Objectives:
• Rationale and advantages of iPSC-derived therapies
• Details of first completed clinical trial worldwide with iPSC-derived cells in any disease
• Overview of broad ongoing clinical development pipeline
• Partnering opportunities
Presenter:
Chief Operating Officer
Cynata Therapeutics Limited
Australia
Stem Cell-derived Exosome Production In Stirred-tank Bioreactors
Available On-Demand on May 26
Learning Objectives:
• Learn about the potential of stem cell-derived exosomes in therapeutic applications
• Understand, how stirred-tank bioreactors facilitate the production of large amounts of exosomes
• Learn, how bioreactors can help increase reproducibility of exosome production
• Get to know the DASbox® Mini Bioreactors System equipped with BioBLU® Single-Use Vessels, which was successfully used for exosome production
Presenter:
Application Specialist-Cell Handling
Eppendorf Application Technologies SA
Belgium
Functional Markers of Bone Marrow Derived Mesenchymal Stem Cells For Orthopedic Applications
Available On-Demand on May 26
Presenter:
AO Research Institute (ARI)
USA
Cell and Gene Therapy Product Quality with the Aura CL
Available On-Demand on May 26
Learning Objectives:
• Learn cell and gene therapy-specific techniques for:
• Particle analysis
• Product purity
• Product quality
Presenter:
Bernardo Cordovez, PhD
Chief Science Officer
Halo Labs
USA
Highly Multiplexed, Single-Cell Functional Proteomics of CAR-T Products Enables More Predictive Product Characterization, Cell Manufacturing Optimization and Cellular Biomarkers across Product Types
Available On-Demand on May 26
We review single-cell polyfunctional profiling results from several different sets of pre-infusion CAR-T samples, including anti-CD19 CAR-T samples from both Kite Pharma (Gilead) [1] and Novartis Pharmaceuticals [2], GoCAR-T cell products from Bellicum Pharmaceuticals [3], and Bispecific CD19/22 CAR-T cells from the NIH [4]. trimeric APRIL-based CAR-T cells targeting both BCMA and TACI from MGH [5].
Presenter:
IsoPlexis
USA
Next Gen Cell Therapy Manufacturing with ekko™ Acoustic Cell Processing System
Available On-Demand on May 26
Learning Objectives:
• The best practices for the industrialization of cell therapy manufacturing
• How acoustic cell processing technology addresses the needs of the entire manufacturing workflow
• How process optimization can be supported through use of the ekko™ Architect software, our protocol development tool for the ekko™ Cell Processing System
Presenters:
Samantha Sbardella
MilliporeSigma
USA
MilliporeSigma
USA
Intelligent Media Design to Drive Better Therapeutic Outcomes
Available On-Demand on May 26
Learning Objectives:
• Single cell-media components can affect transfection efficacy, potency, and yield of your cell therapies.
• Improvement of black-box proprietary off the shelf media formulas, critical for cell-performance, is a manual process requiring a high degree of experimentation and validation.
• Media variability has been demonstrated between suppliers. Modification on top of unwanted variability amplifies the problem. Variability creates non-scalability. Avoid it.
• Design and own your own formulations and eliminate variability, with consistent, high-quality supply of materials to scale with your stage of development.
• Eliminating variability supports scalable volumes, from benchtop to bioreactor.
• Transparency is critical in every stage of cell culture media development from formulation to supply.
Presenters:
Brad Taylor
Nucleus Biologics
USA
Louisiana-Based Clinical Product Manufacturing for Tissue Repair
Available On-Demand on May 26
Learning Objectives:
• To describe optimal features for products that support human tissue modeling and injury repair
• Discuss the value of human adipose-derived matrices for tissue repair applications in skin, bone, and connective tissues
Presenters:
Trivia Frazier, PhD, MBA
Obatala Sciences, Inc.
USA
Allogenic Cell Therapy Cryopreservation Process Development and Optimization
Available On-Demand on May 26
We will demonstrate that:
• Cryopreservation process development is often left to the last minute
• Successful cryopreservation is critical to efficient product development
• Cryopreservation development can be integrated into the continuum of process development
• The required components for a successful cryopreservation process
• Pluristyx can develop and optimize your custom cryopreservation workflow
Presenters:
Brian Hawkins, PhD
Pluristyx
USA
Key Considerations for hMSC Process Development
Available On-Demand on May 26
Learning Objectives:
• Understand RoosterBio’s products and capabilities positioned to enable customers for their hMSC based therapeutic needs
• Insight in RoosterBio’s Upstream and Downstream Process Development for scalable cell production
Presenters:
Theresa D'Souza, PhD
RoosterBio, Inc.
USA
Data-driven Solutions to Understand and Minimize Failures of Cell Collections
Available On-Demand on May 26
150 Million-fold Cumulated Expansion of Encapsulated HIPS Cells in Bioreactor
Available On-Demand on May 26
Learning Objectives:
• Comparing expansion factors & overall scalability of iPS cell culture systems
• Comparing the output of iPS technologies in terms of cell quality
Presenters:
Chief Development Officer
TreeFrog Therapeutics
France
Chief Scientific Officer and Co-Founder
TreeFrog Therapeutics
France
Chief Technical Officer and Co-Founder
Treefrog Therapeutics
France
Vector Construction for Cell and Gene Therapy
Available On-Demand on May 26
Learning Objectives:
•Introduction to VectorBuilder’s intuitive design studio
•Easy design of vectors
•Applications in cell and gene therapy
•Virus production for research and clinical trials
Presenters:
Territory and Business Development Manager
VectorBuilder Inc.
USA
Sr. Territory Manager
VectorBuilder GmbH
Spain
Southeast & TX Sales Representative
VectorBuilder Inc.
USA
WellSky Biotherapies – Simplifying Data Management for Cell and Gene Therapies
Available On-Demand on May 26
Attendees of this showcase will:
•Learn about Biotherapies, WellSky's software solution for cell and gene therapy
•Understand how Biotherapies addresses supply chain management
•Understand solution configurability across donor management and eligibility determination, collection / apheresis, and processing / manufacturing platforms and protocols
• Understand how the solution manages equipment and supply inventory
• Understand label printing (i.e. ISBT-128) capabilities
• Learn how the solution supports clinical trials / research studies
• Understand solution interface capabilities
• Learn how the system makes data actionable
Presenters:
General Manager - Biotherapies
WellSky
USA
How to Build A Cryogenic Logistics Platform for Allogeneic Therapies
Available On-Demand on May 26
Presenter:
AmerisourceBergen World Courier
Senior Director, Personalized Supply Chain
Germany
The Path to a Biologics License Application (BLA)
MAY 26 - 11:30 EDT
Cell and gene therapy treatments often serve rare disease populations. Clinical development includes clinical studies using fewer treated patients than would normally be used for traditional pharmaceutical Phase 1, 2 and 3 studies. These shortened development timelines mean needing to be ready for commercial approval can happen quickly. These series of topics reflect some common areas of risk that must be addressed prior to product approval.
Presenters:
Karen Nichols, Esq.
Vertex Pharmaceuticals
USA
Victoria Dohnal, RAC
Biotechnology Innovation Organization
USA
Dark Horse Consulting
USA
Beam Therapeutics
USA
Michael Mendicino, PhD
Hybrid Concepts International
USA
Panelist
Mo Heidaran, PhD
Paraxel International
USA
Phishing for Cell Therapies - Therapeutic Deception
MAY 26 - 17:00 EDT
ISCT Presidential Task Force (PTF) on the use of Unproven/Unethical Cell & Gene Therapies
Direct-to-consumer (DTC) marketing of unproven and unlicensed cell-based interventions remains a borderless, serious challenge despite concerted efforts to regulate. DTC marketing frequently uses tokens of scientific and clinical legitimacy and includes extravagant claims about products to appeal to patients.
Such strategies may amount to therapeutic deception and are increasingly under scrutiny by regulatory agencies, such as the US Food and Drug Administration (FDA) and Federal Trade Commission (FTC) and the European Medicines Agency (EMA). In this session, we discuss multipronged efforts by different stakeholders, such as tech companies and legislators, to counteract misleading messaging and address the challenges posed by the global reach of the DTC industry.
Learning objectives:
• Understand deceptive practices in direct-to-consumer marketing that targets patients
• Review current efforts at the national and international level to delegitimize and contain such practices
Presenters:
Laertis Ikonomou, PhD
University of Buffalo
USA
The Ohio State University
USA
Vermont State Senate
USA
Federal Trade Commission
USA
Therapeutic Goods Administration
Australia
Understanding the Necessary Conditions for First in Human Trials
MAY 28 - 15:30 EDT
Initiating FIH clinical studies for cell or gene therapy products create some unique challenges, despite being required to meet all regulatory requirements for filing and IND and securing Study May Proceed. These topics provide a comprehensive discussion of several current requirements.
Presenters:
Karen Nichols, Esq.
Vertex Pharmaceuticals
USA
Co-Chair
Karen Major
Turnstone Biologics
Canada
University of Miami
USA
Ensoma
USA
US Pharmacopeia
USA
Global Regulatory Perspectives
MAY 29 - 17:00 EDT
Global Regulatory Perspectives Workshop
This flagship program represents the diverse activities and partnerships between international regulatory bodies, industry, clinicians and academia. This year’s topics include viral vector quality (translating from preclinical to First in Human, viral vectors compliance complexities), a discussion on gene editing components, as well as regulatory CMC issues affecting the clinical development of cell and gene therapies.
Presenters:
Karen Nichols, Esq.
Vertex Pharmaceuticals
USA
Co-Chair
Dominic Wall, PhD, FFSc (RCPA)
Peter MacCallum Cancer Center, Australia
Advanced Cell & Gene Therapy
USA
Thermo Fisher Scientific
USA
Jessica Chery, PhD
FDA
USA
Matthew Klinker, PhD
FDA
USA
ISCT CSO Top Scoring Abstract Showcase
Oral Abstract Presentations:
Antonella Pinto (US) - Derivation of Folliculogenic Organoids from Human iPSC (Abstract 1)
Steve Oh (SG) - Generation Of High Densities Of Universal O-ve Red Blood Cells From Human Induced Pluripotent Stem Cells In Bioreactors (Abstract 6)
Sabiha Hacibekiroglu (CA) – Engineered Safe and Immune-Tolerant ‘Designer’ RPE Cells Towards the Treatment of Age-Related Macular Degeneration (Abstract 5)
Hyunjung Min (US) - Mesenchymal Stromal Cells Reprogram Macrophages with Processing Bodies (Abstract 4)
Andrea Mohr (UK) - Fas-Threshold Signalling in MSCs Causes Tumour Progression and Metastasis (Abstract 3)
Tom Wyrobnik (SG) - Engineering Characterisation of A Novel Impeller For Use In Stirred-tank Bioreactors (Abstract 2)
Chaired By:
Daniel J. Weiss, MD, PhD
University of Vermont
USA
ISCT-AABB Joint Working Group Project Updates
This session will provide an overview and updates on two current projects.
The Stability Project:
This project aims to educate the community regarding consideration for cellular therapy product stability programs. The deliverable will be to reference documents and/or other communication materials to help with the design of a stability program for HPC products specifically.
GTP Interpretative Tool Project:
This project aims to establish a GTP interpretative tool that can assist with clarification of HCT/P regulations under 21 CFR 1271 and Section 361 of the PHS Act and/or the FD&C Act
Presenters:
Michele Sugrue, MS, MT (ASCP) SBB
University of Florida
USA
University of Illinois Cancer Centre
USA
National Institutes of Health
USA
Impact of COVID-19 on the Practice of the CTL: Global Perspectives
The COVID-19 pandemic brought numerous challenges to the Cellular Therapy community including implementation of additional testing requirements for both donors & recipients, overcome travel restrictions both locally, nationally, and internationally and implementation of new processes such as universal cryopreservation. The session will address how the cell therapy community addressed these challenges to best provide for the patients’ needs.
The session will describe the actions taken by NMDP/Be The Match to adapt to the evolving situation. In addition –different perspectives of cell therapy facilities across the world will be presented describing the changes in their respective practices.
Presenters:
Joseph Schwartz, MD, MPH
New York Presbyterian Hospital
USA
Vicki Antonenas, BSc, MSc
Westmead Hospital
Australia
Columbia University
USA
NHS Blood and Transplant
UK
NMDP / Be The Match
USA
Lessons in MSC Applications from Pet Populations with Naturally Occurring Diseases
While cell-based therapy holds tremendous promise due to therapeutic efficacy evidenced in early stage clinical development, products reaching the market remains scarce, often due to failure of late-stage clinical trials. Strategies to optimize successful translation of cell-based therapies include collection of more predictive pre-clinical data through use of optimized pre-clinical models, defined mode(s) of action (MoA), and robust potency assays in each proposed indication. Humans and domestic animals are in close contact, share the same environments, and develop similar chronic inflammatory diseases. This session will discuss how data obtained from pre-clinical animal models or animal populations with naturally occurring disease can be valorized to improve research outcomes for both veterinary and human medicine.
Learning Objectives: Data obtained from regenerative medical research using strong pre-clinical animal models or animal populations with naturally occurring disease will highlight similarities and peculiarities of cell-based approaches in human and veterinary studies. Examples of both immune-mediated pathologies (i.e. atopic dermatitis, vasculitis, …) and inflammatory osteoarthritis will be covered. The information provided will assist researchers in determining additional ways to advance translational regenerative medicine through collaboration and use of strategic pre-clinical trials.
Presenters:
Tracy Webb, DVM, PhD
Colorado State University
USA
Antonio Villatoro, DVM, PhD, MBA
ImmuneStem
Spain
Vetbiobank
France
COVID-19 Therapies and Complications of CAR T-cell therapy
This session will discuss non-pharmacologic and pharmacologic treatment for COVID-19 infection.
We will also discuss the management of neurotoxicity, as well as long-term complications and survivorship post CAR T-Cell therapy.
Presenters:
Nicholas Szewczyk, APRN, MSN, ANP-C
University of Texas MD Anderson Cancer Center
USA
Jeremy Randial, MD
MD Anderson Cancer Center
USA
Memorial Sloan Ketter