Orchestrating Global Cell and Gene Therapy Translation
Building Consensus for the Path Forward
PLENARY PROGRAM
An ensemble of global experts gathered from academic, regulatory, and industry perspectives to build consensus on the key issues lying ahead on the path forward
Hover over session chairs to see what they have to say, and over speakers to learn who they are.
PLENARY I
COVID-19: Understanding the Path Forward for Mesenchymal Stromal Cell Therapies
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.
“There is a diversity in the field regarding dosing and timing of administration in clinical trajectory.
…It remains to be defined which allogeneic tissue source of MSCs deploys cellular attributes foreshadowing a biological and clinical response, as well as subject predictive markers of response”
Session Chair
Jacques Galipeau, MD
University of Wisconsin-Madison
USA
Maroun Khoury, PhD
Cells for Cells – REGENERO
Chile
Patricia Rocco, MD, PhD
Federal University of Rio de Janeiro
Brazil
Anthony Ting, PhD
Athersys
USA
PRESIDENTIAL PLENARY
Integrating Science, Regulatory Oversight, and Commercialization for the New Wave of Cell & Gene Therapies
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.
Our speakers have each demonstrated a keen ability, in their respective areas, to adapt to the unexpected… Each of them is a proven leader in their respective sphere of regulatory, commercial, and academic expertise, strengthened by experience across other parts of the translational chain.
Session Chair
Bruce Levine, PhD
University of Pennsylvania
USA
Arie Belldegrun, MD
Allogene Therapeutics
USA
Peter Marks, MD, PhD
FDA Center for Biologics Evaluation and Research
USA
Matthew Porteus, MD, PhD
Stanford University
USA
PLENARY III
“First in Man” Exosome Trials
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.
“Scientific rationales and generation of evidence necessary for proof-of-concept are key considerations in developing human clinical trials. Collaborative translation of preclinical data will inform successful development of exosomes into therapeutics”
Session Co-Chair
Sai-Kiang Lim, PhD
Institute of Molecular & Cell Biology
Singapore
Eva Rohde, MD
Paracelsus Medical University
Austria
Andrew Hoffman, DVM, DVSc
University of Pennsylvania
USA
Session Co-Chair
Maurizio Muraca, MD, PhD
University of Padova
Italy
“It will be critical to seek consensus on issues such as translating effective dosage from animal to human studies, quantifying EV drug preparation, investigating toxicity, and investigating the MoA to validate in vitro potency assays.”
PLENARY IV
BMT vs Gene Therapy vs No Cell Therapy:
Accessibility, Cost, and Outcomes
We begin 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…
“…the panel will carefully consider and weigh a host of factors that impact the chances of success of their respective approaches. Acknowledging that things evolve over time will be important in building an informative consensus…”
Session Chair
Jaap Jan Boelens, MD, PhD
Memorial Sloan Kettering
USA
Alexis Thompson, MD, MPH
Ann & Robert H. Lurie Children’s Hospital of Chicago
United States
Suradej Hongeng, MD
Ramathibodi Hospital, Mahidol University
Thailand
Selim Corbacioglu, MD
University Children’s Hospital Regensburg
Germany
Matthew Porteus, MD, PhD
Stanford Medicine
USA
Deepa Manwani, MD
The Children’s Hospital at Montefiore
USA
Session Chair
Sandeep Soni, MD
CRISPR Therapeutics
USA
“…seeking consensus is not necessarily to endorse one approach as a field… consensus is a decision to collaborate across the field to promote continued impactful research in light of rapid developments…”
PLENARY V
Charting the Future – Safe and Effective Translation in Tissue Engineering and Regenerative Medicine
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.
Session Chair
Ranieri Cancedda, MD
Università degli Studi di Genova
Italy
Anthony Atala, MD
Wake Forest Institute for Regenerative Medicine
USA
Graziella Pellegrini, PhD
University of Modena and Reggio Emilia
Italy
Rocky Tuan, PhD
Chinese University of Hong Kong
Hong Kong
Session Chair
Giuseppe Orlando, MD, PhD
Wake Forest University
USA
“Eminent speakers will guide us through the current landscape of tissue engineering and regenerative medicine, illustrate the state of the art, and integrate lessons from stem cell therapies and 3D printing to propose a path forward towards safe translation”
PLENARY VI
Debating Applications of CAR-T/NK cells:
Perspectives on Autologous versus Allogeneic Therapies
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.
“Progress is contingent on addressing issues related to manufacturing, efficacy, regulatory approvals, cost, patient access, pricing, and availability of therapies to underserved populations. Consensus on these issues provides critical information for decisions on optimal uses of cellular immunotherapy sources.”
Session Chair
Michael Gustafson, PhD
Mayo Clinic Arizona
USA
Rayne Rouce, MD
Baylor College of Medicine
USA
Wen Bo Wang, PhD
Fate Therapeutics
USA
PLENARY I
COVID-19: Understanding the Path Forward for Mesenchymal Stromal Cell Therapies
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.
“There is a diversity in the field regarding dosing and timing of administration in clinical trajectory.
...It remains to be defined which allogeneic tissue source of MSCs deploys cellular attributes foreshadowing a biological and clinical response, as well as subject predictive markers of response”
Session Chair
Jacques Galipeau, MD
University of Wisconsin-Madison
USA
Maroun Khoury, PhD
Cells for Cells - REGENERO
Chile
Patricia Rocco, MD, PhD
Federal University of Rio de Janeiro
Brazil
Anthony Ting, PhD
Athersys
USA
PRESIDENTIAL PLENARY
Integrating Science, Regulatory Oversight, and Commercialization for the New Wave of Cell & Gene Therapies
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.
Our speakers have each demonstrated a keen ability, in their respective areas, to adapt to the unexpected... Each of them is a proven leader in their respective sphere of regulatory, commercial, and academic expertise, strengthened by experience across other parts of the translational chain.
Session Chair
Bruce Levine, PhD
University of Pennsylvania
USA
Arie Belldegrun, MD
Allogene Therapeutics
USA
Peter Marks, MD, PhD
FDA Center for Biologics Evaluation and Research
USA
Matthew Porteus, MD, PhD
Stanford University
USA
PLENARY III
“First in Man” Exosome Trials
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.
"Scientific rationales and generation of evidence necessary for proof-of-concept are key considerations in developing human clinical trials. Collaborative translation of preclinical data will inform successful development of exosomes into therapeutics"
Session Co-Chair
Sai-Kiang Lim, PhD
Institute of Molecular & Cell Biology
Singapore
Eva Rohde, MD
Paracelsus Medical University
Austria
Andrew Hoffman, DVM, DVSc
University of Pennsylvania
USA
Session Co-Chair
Maurizio Muraca, MD, PhD
University of Padova
Italy
"It will be critical to seek consensus on issues such as translating effective dosage from animal to human studies, quantifying EV drug preparation, investigating toxicity, and investigating the MoA to validate in vitro potency assays."
PLENARY IV
BMT vs Gene Therapy vs No Cell Therapy:
Accessibility, Cost, and Outcomes
We begin 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…
“...the panel will carefully consider and weigh a host of factors that impact the chances of success of their respective approaches. Acknowledging that things evolve over time will be important in building an informative consensus..."
Session Chair
Jaap Jan Boelens, MD, PhD
Memorial Sloan Kettering
USA
Alexis Thompson, MD, MPH
Ann & Robert H. Lurie Children's Hospital of Chicago
United States
Suradej Hongeng, MD
Ramathibodi Hospital, Mahidol University
Thailand
Selim Corbacioglu, MD
University Children's Hospital Regensburg
Germany
Matthew Porteus, MD, PhD
Stanford Medicine
USA
Deepa Manwani, MD
The Children's Hospital at Montefiore
USA
Session Chair
Sandeep Soni, MD
CRISPR Therapeutics
USA
"...seeking consensus is not necessarily to endorse one approach as a field... consensus is a decision to collaborate across the field to promote continued impactful research in light of rapid developments..."
PLENARY V
Charting the Future – Safe and Effective Translation in Tissue Engineering and Regenerative Medicine
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.
Session Chair
Ranieri Cancedda, MD
Università degli Studi di Genova
Italy
Anthony Atala, MD
Wake Forest Institute for Regenerative Medicine
USA
Graziella Pellegrini, PhD
University of Modena and Reggio Emilia
Italy
Rocky Tuan, PhD
Chinese University of Hong Kong
Hong Kong
Session Chair
Giuseppe Orlando, MD, PhD
Wake Forest University
USA
"Eminent speakers will guide us through the current landscape of tissue engineering and regenerative medicine, illustrate the state of the art, and integrate lessons from stem cell therapies and 3D printing to propose a path forward towards safe translation"
PLENARY VI
Debating Applications of CAR-T/NK cells:
Perspectives on Autologous versus Allogeneic Therapies
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.
“Progress is contingent on addressing issues related to manufacturing, efficacy, regulatory approvals, cost, patient access, pricing, and availability of therapies to underserved populations. Consensus on these issues provides critical information for decisions on optimal uses of cellular immunotherapy sources.”
Session Chair
Michael Gustafson, PhD
Mayo Clinic Arizona
USA
Rayne Rouce, MD
Baylor College of Medicine
USA
Wen Bo Wang, PhD
Fate Therapeutics
USA
