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ISCT-ABTCel-Gen Joint Webinar: Mitochondria Transfer and Transplant: Powering the Cell Therapy Field
WEBINAR DESCRIPTION: In October 2023, the FDA issued a guidance document on Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies. This guidance describes a standards recognition program for regenerative medicine therapies (SRP-RMT) at FDA’s Center for Biologics Evaluation and Research (CBER) designed to identify and recognize Voluntary Consensus Standards (VCS) to facilitate the development and assessment of regenerative medicine therapy (RMT) products regulated by CBER when such standards are appropriate. Standardization bodies such as Standards Coordinating Body (SCB) and the American National Standards Institute (ANSI) have helped to introduce and facilitate the VCS process, however, many stakeholders are unclear about the process. This webinar will discuss the VCS process, what the stakeholders need to know, and how to develop one. A case study of an actual international consensus standard development of tissue-specific MSC biobanking, characterization, terminology with extensive and iterative input from ISCT will also be presented.
Presented by: ISCT South and Central America Regional Executive Committee
November 20, 2024
Webinar Description:
Join us for an insightful journey into the evolving field of mitochondrial transfer and transplant with our webinar titled "Mitochondria Transfer and Transplant: Powering the Cell Therapy Field. Co-organized by the ISCT South & Central America (SCA) Regional Executive Committee and Associação Brasileira de Terapia Celular e Gênica (ABTCel-Gen), the event will be co-chaired by Virginia Picanço-Castro, PhD and Andrés Caicedo, PhD.
The webinar will open with Andrés Caicedo providing an introduction to the field and key insights from his laboratory team's pioneering research on mitochondrial transfer in skin cells and its application in wound healing and tissue regeneration. He will emphasize how mitochondria have evolved from being seen as mere powerhouses of the cell to “Living Drugs” with immense therapeutic potential.
Our featured speaker, Jonathan Brestoff, MD, PhD, will present his groundbreaking research on mitochondria transfer between adipocytes and macrophages, showcasing the intersection of immunometabolism and mitochondrial biology. Maroun Khoury, PhD, will delve into the revolutionary use of mitochondria transfer in CAR T cell therapies, highlighting its potential to transform cancer treatment.
This exclusive event offers members of the International Society for Cell & Gene Therapy (ISCT) an opportunity to explore how mitochondria are driving advances in cell therapy, addressing key challenges in immune dysfunction, and tissue repair. Engage with leading experts and discover how mitochondrial research is reshaping the future of regenerative medicine.
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WEBINAR DESCRIPTION: In October 2023, the FDA issued a guidance document on Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies. This guidance describes a standards recognition program for regenerative medicine therapies (SRP-RMT) at FDA’s Center for Biologics Evaluation and Research (CBER) designed to identify and recognize Voluntary Consensus Standards (VCS) to facilitate the development and assessment of regenerative medicine therapy (RMT) products regulated by CBER when such standards are appropriate. Standardization bodies such as Standards Coordinating Body (SCB) and the American National Standards Institute (ANSI) have helped to introduce and facilitate the VCS process, however, many stakeholders are unclear about the process. This webinar will discuss the VCS process, what the stakeholders need to know, and how to develop one. A case study of an actual international consensus standard development of tissue-specific MSC biobanking, characterization, terminology with extensive and iterative input from ISCT will also be presented.
Presented by: ISCT North America LRA Committee
November 19, 2024
Webinar Description:
In October 2023, the FDA issued a guidance document on Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies. This guidance describes a standards recognition program for regenerative medicine therapies (SRP-RMT) at FDA’s Center for Biologics Evaluation and Research (CBER) designed to identify and recognize Voluntary Consensus Standards (VCS) to facilitate the development and assessment of regenerative medicine therapy (RMT) products regulated by CBER when such standards are appropriate.
Standardization bodies such as Standards Coordinating Body (SCB) and the American National Standards Institute (ANSI) have helped to introduce and facilitate the VCS process, however, many stakeholders are unclear about the process.
This webinar will discuss the VCS process, what the stakeholders need to know, and how to develop one. A case study of an actual international consensus standard development of tissue-specific MSC biobanking, characterization, terminology with extensive and iterative input from ISCT will also be presented.
J. Wade Atkins, MS, MLS,SBB (ASCP), CQA , CABP(H)
Supervisor, QA and Regulatory Affairs; Member of ISCT North America Legal & Regulatory Affairs Committee
J. Wade Atkins is an MT ASCP with a Specialty in Blood Banking, is credential as a Certified Advanced Biotherapies Professional, a certified Quality Auditor through the American Society for Quality and has a Master of Science in Health Care Administration.
For 22 years he has been associated with quality assurance and regulatory affairs for the Department of Transfusion Medicine (DTM) in the Clinical Center for the National Institutes of Health in Bethesda Maryland and is currently supervising a staff of 9 quality associates. He has responsibilities for ensuring compliance with standards and regulations for manufacturing high quality products.
The DTM is a full-service blood bank with a licensed collection facility and a full transfusion service and maintains an active HCTP manufacturing facility that supports roughly 80 protocols and 35 or more of those are regulated by IND at the FDA. The Blood establishment and Center for Cellular Engineering employee about 200 staff.
Catherine (Katie) Zander, PhD
Director of Standards Education & Outreach
Standards Coordinating Body
In her role with SCB, Katie brings a combination of leadership and technical skills gained from her prior experience as a researcher, a postdoctoral community advocate, and a scientific policy advisor in Congress.
Prior to joining SCB, Katie worked in U.S. House of Representatives for the Committee on Energy and Commerce as the American Society of Hematology’s inaugural American Association for the Advancement of Science (AAAS) Science & Technology Policy Fellow. Before the fellowship, she conducted research on rare blood clotting diseases at the University of Alabama at Birmingham, where she also founded a patient education program. She holds a PhD in Chemistry from Binghamton University, where she studied neurotransmitter kinetics.
Christine Aston-Roberts
Standards Manager, Senior
Parenteral Drug Association (PDA)
Christine joined the Parenteral Drug Association (PDA) as their Senior Standards Manager in 2017. Prior to this, she was affiliated with the American Type Culture Collection where over 16 years, she served in numerous capacities, to include technical services, a cell biology lab, and as a standards and certification specialist. She served for 13 years in clinical transplant in the histocompatibility lab at the Medical College at Virginia in Richmond, Virginia, Sentara Norfolk General Hospital in Norfolk, Virginia. She also worked as a Senior Donor Services Coordinator for the “Be The Match” program operated by the National Marrow Donor Program/ CW Bill Young Marrow Program in the Washington DC area. She earned her degree from Virginia Tech.
Sowmya Viswanathan, PhD
Scientist, Osteoarthritis Program, Division of Orthopedic Surgery, Schroeder Arthritis Institute and the Krembil Research Institute; ISCT North America Regional Vice-President Elect
University Health Network
Dr. Viswanathan is a Scientist at the Osteoarthritis Program, Division of Orthopedic Surgery, Schroeder Arthritis Institute and the Krembil Research Institute (University Health Network) and an Associate Professor at the Institute of Biomedical Engineering and at the Division of Hematology, Department of Medicine (University of Toronto).
Dr. Viswanathan is leading international efforts to standardize MSC nomenclature and create reference materials for MSC research. Dr. Viswanathan sits on the Standard Council of Canada’s Mirror Committee for International Organization for Standardization (ISO TC276) on Analytical Methods and Bioprocessing, and is on the steering and working committee of an international Standards Coordinating Body (SCB). Dr. Viswanathan is a formal liaison between ISO TC276 and the International Society of Cell and Gene Therapy (ISCT). Dr. Viswanathan is Associate Editor of Cytotherapy, the official journal for ISCT and N. American VIP Elect (2024-2026) for ISCT.
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WEBINAR DESCRIPTION: Join us for a deep dive into the world of CAR-T cell therapy development with our webinar titled "Navigating CAR-T Cell Therapy Development: Insights from Leading SCA Academic Centers." Organized by the ISCT South & Central America (SCA) Regional Executive Committee and the South and Central America (SCA) Regional ESP Subcommittee. The event will be co-chaired by Samuel Couto and Lilia León Moreno. The webinar will open with Virginia Picanço-Castro sharing her experience from one of Brazil's leading institutes on the complete developmental journey of a CAR-T cell product, from bench to bedside. She will provide a detailed overview of the unique manufacturing challenges faced and the innovative strategies implemented to bring CAR-T cell therapies to clinical practice in Brazil. Andres Gomez will offer valuable insights from Mexico, discussing the development and implementation of CAR-T cell therapies within the local academic and clinical landscape. He will highlight the regulatory environment and the steps required to advance this novel treatment in Mexico. Chase Mccann will conclude the session by discussing the role of clinical trials in the development of CAR-T cell therapies, drawing from his experience at Children's National Hospital. He will focus on the latest advancements in pediatric applications and share real-world insights from ongoing clinical trials. This exclusive event offers members of the International Society for Cell & Gene Therapy (ISCT) an opportunity to explore the diverse paths of CAR-T therapy development across the South & Central America region. Engage with leading experts and discover the future of CAR-T therapy, from clinical trials to regulatory challenges and beyond.
Presented by: ISCT South and Central America Regional Executive Committee & South & Central America Regional ESP Subcommittee
November 5, 2024
Webinar Description:
Join us for a deep dive into the world of CAR-T cell therapy development with our webinar titled "Navigating CAR-T Cell Therapy Development: Insights from Leading SCA Academic Centers." Organized by the ISCT South & Central America (SCA) Regional Executive Committee and the South and Central America (SCA) Regional ESP Subcommittee. The event will be co-chaired by Samuel Couto and Lilia León Moreno.
The webinar will open with Virginia Picanço-Castro sharing her experience from one of Brazil's leading institutes on the complete developmental journey of a CAR-T cell product, from bench to bedside. She will provide a detailed overview of the unique manufacturing challenges faced and the innovative strategies implemented to bring CAR-T cell therapies to clinical practice in Brazil.
Andres Gomez will offer valuable insights from Mexico, discussing the development and implementation of CAR-T cell therapies within the local academic and clinical landscape. He will highlight the regulatory environment and the steps required to advance this novel treatment in Mexico.
Chase Mccann will conclude the session by discussing the role of clinical trials in the development of CAR-T cell therapies, drawing from his experience at Children's National Hospital. He will focus on the latest advancements in pediatric applications and share real-world insights from ongoing clinical trials.
This exclusive event offers members of the International Society for Cell & Gene Therapy (ISCT) an opportunity to explore the diverse paths of CAR-T therapy development across the South & Central America region. Engage with leading experts and discover the future of CAR-T therapy, from clinical trials to regulatory challenges and beyond.
Samuel Campanelli Freitas Couto, PhD
Coordinator of the Cell Therapy Laboratory at the Blood Center of São Paulo and researcher at University of São Paulo.
São Paulo Blood Center (Fundação Pró-Sangue)
Samuel C. F. Couto, PhD, is a researcher at the Blood Center of São Paulo (Fundação Pró-Sangue) and also serves as an associate researcher at the University of São Paulo in Brazil. After earning his degree in Biological Sciences from PUC-Campinas University, Dr. Couto pursued a PhD in Biotechnology at the University of São Paulo, followed by postdoctoral fellowships at both the University of São Paulo's Medical School and Children’s National Hospital in Washington, D.C. He is particularly interested in increasing access to cellular and gene therapies in lower-middle-income countries. He also leads the Laboratory of Stem Cell Processing at the University of São Paulo’s Clinics Hospital (HC/FMUSP).
Lilia Carolina León-Moreno, PhD
Posdoctoral Researcher
Centro de Investigación y Asistencia en Tecnología y Diseño del Estado de Jalisco
Lilia C. León-Moreno, PhD, is a posdoctoral researcher focused on the study of exosomes and mesenchymal stromal cells as a potential therapy for neurodegenerative diseases. She is interested in increasing the knowledge of the medical and scientific communities, and general public about advanced therapies and establishing a regulatory framework that enables the development of these therapies in Latin America. She is currently involved in the planning of a CDMO for cell therapy in Jalisco.
Virginia Picanço e Castro, PhD
Coordinator of the Biotechnology Laboratory at the Ribeirão Preto Blood Center and researcher at the CTC-USP
Ribeirão Preto Blood Center
Virginia Picanço e Castro, PhD, is a prominent researcher at the Regional Blood Center of Ribeirão Preto in Brazil, where she focuses on pluripotent stem cells and cell reprogramming. After earning her degree in Biological Sciences from the University of São Paulo, Dr. Picanço e Castro pursued a Ph.D. in Genetics at the Medical School of Ribeirão Preto, followed by postdoctoral fellowships at both the Regional Blood Center and Indiana University. Her research explores the complex pathways involved in cell reprogramming, specifically aiming to generate pluripotent stem cells from human somatic cells. She also investigates the molecular mechanisms that regulate the differentiation of hematopoietic and pluripotent stem cells, with the ultimate goal of developing effective in vitro differentiation protocols.
Dr. Picanço e Castro’s work aims to create a comprehensive database of genomic proto-proteins involved in hematopoietic differentiation, which could lead to new gene overexpression techniques that enhance cell differentiation and reprogram fibroblasts into adult blood cells. Her research is highly relevant to Brazil's burgeoning biotechnology sector, with the potential to offer safer and more efficient stem cell-based therapies. She collaborates with leading national and international researchers and guides a team of students at the Biotechnology Laboratory Group, contributing to significant advancements in regenerative medicine and therapeutic innovation.
Andrés Gómez-De León, MD
Associate Professor of Hematology
Universidad Autónoma de Nuevo León
Andrés Gomez-De Léon, MD, is based in a University Hospital in Mexico and focuses on acute leukemia and hematopoietic stem cell transplantation. He is committed to enhancing healthcare access in low-resource settings, aiming to provide high-quality, cost-effective care. His institution is Mexico's largest transplant center, known for its pioneering work in outpatient allogeneic transplants. A graduate and faculty member of the American Society of Hematology Clinical Research Training Institute, he collaborates globally, drives clinical trials in leukemia, and builds a diverse research team. He also creates educational content in Spanish to promote hematology research and training across borders.
Chase D. McCann, PhD, MSPH
Assistant Director of Manufacturing
Children’s National Hospital
Chase D. McCann, PhD, MSPH, is an Assistant Research Professor of Pediatrics at The George Washington University School of Medicine and Health Sciences and the Assistant Director of Manufacturing at Children's National Hospital Cell Therapy Laboratory. He completed his Ph.D. in Immunology and Microbial Pathogenesis at Weill Cornell in 2020, following an M.S. in Public Health Microbiology from The George Washington University and a BSc in Biology and Neuroscience Psychology from UMass Amherst. His professional journey includes leadership roles in cell therapy and research positions at top institutions such as the University of North Carolina and Weill Cornell Medicine. Dr. McCann's research focuses on immune system mechanisms and cell therapy advancements, contributing to significant developments in pediatric and public health immunology.
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WEBINAR DESCRIPTION: While an increasing number of regulatory approvals in the Cell and Gene Therapy (CGT) field has fueled optimism for potential treatments and cures for debilitating and fatal conditions, constant challenges to US FDA oversight – by courts, federal and state legislatures, and rogue “stem cell clinics” - pose serious risks for individual patients and for the entire industry. This webinar begins with an overview of the U.S. legal landscape governing development and therapeutic usage of stem cell and gene therapies, including several recent, highly-consequential court rulings impacting the FDA and other federal agencies. The webinar then addresses recent state developments that have attracted little public attention, including new iterations of “Right-To-Try” and “Medical Freedom” laws, and their legal and practical impact on experimental stem cell and gene therapies. After exploring the wide-spread availability of unproven stem cell and regenerative interventions currently being offered by stem cell clinics and in other unregulated settings, the webinar will highlight existing risks for individual patients and the entire CGT sector, and ways these may be further exacerbated in the near future. The area of unproven stem cell and regenerative medicine clinics will be discussed in more depth as an exemplar of complex legal, policy and ethical challenges to federal and state regulators. The webinar will conclude with an interactive panel discussion and an open Q&A for attendees to raise issues and contribute to the discussion.
Presented by: ISCT Committee on the Ethics of Cell and Gene Therapy (ECGT)
October 29, 2024
Webinar Description:
While an increasing number of regulatory approvals in the Cell and Gene Therapy (CGT) field has fueled optimism for potential treatments and cures for debilitating and fatal conditions, constant challenges to US FDA oversight – by courts, federal and state legislatures, and rogue “stem cell clinics” - pose serious risks for individual patients and for the entire industry. This webinar begins with an overview of the U.S. legal landscape governing development and therapeutic usage of stem cell and gene therapies, including several recent, highly-consequential court rulings impacting the FDA and other federal agencies. The webinar then addresses recent state developments that have attracted little public attention, including new iterations of “Right-To-Try” and “Medical Freedom” laws, and their legal and practical impact on experimental stem cell and gene therapies. After exploring the wide-spread availability of unproven stem cell and regenerative interventions currently being offered by stem cell clinics and in other unregulated settings, the webinar will highlight existing risks for individual patients and the entire CGT sector, and ways these may be further exacerbated in the near future. The area of unproven stem cell and regenerative medicine clinics will be discussed in more depth as an exemplar of complex legal, policy and ethical challenges to federal and state regulators. The webinar will conclude with an interactive panel discussion and an open Q&A for attendees to raise issues and contribute to the discussion. Beth Roxland, JD, M. Bioethics
Senior Advisor on Law, Policy and Bioethics
Roxland Consultants Ltd., The Roxland Law Firm
Beth E. Roxland, J.D., M.Bioethics is a seasoned Attorney & Bioethicist with multifaceted experience across Industry, Law, Government & Academia. She serves as the Senior Advisor on Law, Policy and Ethics for Roxland Consultants Ltd., an independent consultancy where she leverages her unique expertise to provide comprehensive yet practical strategic advice and support to life-science, biotechnology & research entities, law firms, hospitals & healthcare practitioners, venture capital & financial institutions, and professional & patient associations confronting complex legal, ethical, operational & communications challenges in the scientific and medical arenas. Roxland is also a practicing attorney, specializing in litigation, regulation, torts, health law, public health & pandemics, and risk & crisis management. Roxland is a frequent public speaker & author, serves on several academic, medical and research oversight boards, and occupies leadership roles in professional societies.
Roxland’s executive experience includes her prior roles as:
- Johnson & Johnson's Bioethics and Strategy Leader, in the Global Chief Medical Officer's Office;
- Executive Director of The New York State Task Force on Life & the Law;
- Special Advisor to the Commissioner of Health on Stem Cell Research Ethics;
- Senior Litigation Associate at Simpson Thacher & Bartlett LLP;
- Federal Judicial Law Clerk in the Southern District of New York (S.D.N.Y.); and
- Adjunct Professor of Law at NYU School of Law.
Roxland graduated from Columbia University with a Bachelors degree in Biology, and from the University of Pennsylvania Magna Cum Laude with joint degrees in Law and Bioethics (Juris Doctorate – Masters in Bioethics).
Kirstin Matthews, PhD
Fellows in Science and Technology Policy
Rice University's Baker Institute for Public Policy
Kirstin R.W. Matthews, Ph.D., is a fellow in science and technology policy at Rice University’s Baker Institute for Public Policy and a lecturer in the Department of BioSciences at Rice University. She is also a track advisor for the Wiess School of Natural Sciences’ Professional Science Master in Biosciences and Health Policy, a core member of the Rice Synthetic Biology Institute, and a steering committee member for Rice’s Medical Humanities Research Institute.
Matthews is the director of the Baker Institute Science and Technology Policy Program and the Center for Health and Biosciences’ Biomedical Research Program. Her research focuses on ethical and policy issues at the intersection between traditional biomedical research and public policy. Specifically, she focuses on regulation and ethical issues associated with emerging biotechnology, including vaccines, stem cells, synthetic biology, and genomic medicine. Matthews also collaborates with Kenneth Evans and Neal Lane to understand how scientific advice is used in and provided for the federal government, including the White House Office of Science and Technology Policy (OSTP) and the President’s Council of Advisors on Science and Technology (PCAST).
Matthews has a B.A. in biochemistry from The University of Texas at Austin and a Ph.D. in molecular biology from The University of Texas Health Science Center at Houston.
Paul Knoepfler, PhD
Professor, Department of Cell Biology and Human Anatomy
UC Davis Health, School of Medicine
Paul Knoepfler is an award-winning scientist who is particularly interested in what controls stem and cancer cell biological behavior.
Known also for his popular blog – ipscell.com – and passionate stem cell advocacy, Knoepfler and his laboratory team focus on how a cell’s pluripotency control machinery goes awry during cancer.
Knoepfler is using cutting-edge genomics technology to better understand why stem cells behave the way they do, and working to determine how cell behavior can be directed toward safe and effective clinical use as well as to develop new cancer therapies for kids.
He is the author of three books including Stem Cells: An Insider’s Guide and has a popular TED talk on CRISPR and designer babies.
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In Conversation with Asia CGT Startups
In Conversation with Asia CGT Startups
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In Conversation with CGT Leaders
In Conversation with CGT Leaders
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Hemophilia is an inherited bleeding disorder characterized by the deficiency or absence of clotting factors VIII (Hemophilia A) or IX (Hemophilia B). This lack of clotting factors leads to uncontrolled bleeding, which can result in severe health issues, particularly in the joints and brain. Despite significant advances in preventing bleeding episodes and managing the disease through prophylaxis and non-factor products, several needs remain unmet. One critical issue is the lack of a cure for the disease. This challenge is being strategically addressed through the development of innovative cell and gene therapies. The objective of this webinar, led by two eminent researchers, Dr. Edward Tuddenham (UK) and Dr. Blair Gage (Canada), is to discuss the latest advances and challenges associated with the use use of both therapies in treating Hemophilia A and B. Their goal is to achieve a durable reduction in both bleeding risks and the need for exogenous factor administrations.
Presented by the ISCT Gastro-Intestinal Committee
September 13, 2024
Webinar Description:
Hemophilia is an inherited bleeding disorder characterized by the deficiency or absence of clotting factors VIII (Hemophilia A) or IX (Hemophilia B). This lack of clotting factors leads to uncontrolled bleeding, which can result in severe health issues, particularly in the joints and brain. Despite significant advances in preventing bleeding episodes and managing the disease through prophylaxis and non-factor products, several needs remain unmet. One critical issue is the lack of a cure for the disease. This challenge is being strategically addressed through the development of innovative cell and gene therapies. The objective of this webinar, led by two eminent researchers, Dr. Edward Tuddenham (UK) and Dr. Blair Gage (Canada), is to discuss the latest advances and challenges associated with the use use of both therapies in treating Hemophilia A and B. Their goal is to achieve a durable reduction in both bleeding risks and the need for exogenous factor administrations.
- Learn on strategies forecasting the development and clinical application of CGT
- Increase the knowledge and education on the clinical development of CGT
- Showcase hemophilia as significant case example for the evaluation of emerging CGT
- Understand the science of CGT development for hemophilia
Adriana Migliorini, PhD (Moderator)
Senior Post Doctoral Research Fellow
McEwen Stem Cell Institute, University Health Network
Dr. Adriana Migliorini is a senior postdoctoral research fellow in Dr. Maria Cristina Nostro’s laboratory, at the McEwen Stem Cell Institute, University Health Network, Toronto, Canada. She studied medical biotechnology at the University of Florence (Italy) and completed her Ph.D. in human biology, at the Ludwig Maximilian University in Munich, Germany. Her research primarily revolves around the field of human pluripotent stem cells (hPSCs) and developmental biology, with a specific focus on the pancreas. Her current work involves investigating the composition and phenotype of the fetal pancreatic immune niche to gain a deeper understanding of its role during endocrine development and to explore novel applications for the treatment of Type 1 Diabetes (T1D).
Mustapha Najimi, PhD (Moderator)
Director of Research
UCLouvain
Mustapha Najimi is director of research at the institute of Experimental and Clinical Research (IREC) of the Faculty of Medicine and Pharmacy-UCLouvain in Brussels. He holds a PhD in cell and molecular biology from the Pierre & Marie Curie University, Paris in 1999. He joined the team of Pr Etienne Sokal in 2003 to set up a platform of liver cells’ culture dedicated to the development of a clinical cell therapy program. Since then, he led the stem cell group and coordinated the cell culture technology platform of the laboratory of pediatric hepatology and cell therapy. Thanks to an accumulated know-how and significant knowledge of human liver cells’ biology as well as the strategies to isolate them, his research activity led to the discovery of a population of liver mesenchymal progenitor cells. Those cells were the subject of more than 40 international publications, more than 200 citations and the attribution of 7 related patents. Mustapha NAJIMI has actively been/is involved in i) the design and supervision of the large-scale production of the first batches of these progenitor cells, that have been infused to patients with liver diseases at Saint-Luc hospital- Brussels, ii) the technology transfer of this cell therapy product to a spinoff company, iii) the supervision of R&D activities related to those cells with an industrial vision. Thanks to an active network of international collaborations, he continues conducting his research investigations with the major objectives of better understanding the mechanisms governing liver regeneration and improving the use of cell therapy for liver defects.
Blair Gage, PhD
Scientist, Assistant Professor
Ottawa Hospital Research Institute, University of Ottawa
hPSC-derived Liver Endothelium: A potential Cell Therapy for Hemophilia A
Blair K. Gage (PhD) is a Scientist at the Ottawa Hospital Research Institute in the Regenerative Medicine Program and an Assistant Professor in the Department of Cellular and Molecular Medicine at the University of Ottawa. After a BSc in Biotechnology from UBC and BCIT, he completed a PhD in Cell and Developmental Biology exploring how transcription factors regulated pancreatic endocrine subtype specification from human pluripotent stem cells. His postdoctoral work shifted to generate organ-specific endothelial cells of the liver from human pluripotent stem cells to create potential cell-based and cell-informed therapeutics for liver disease and Hemophilia A. Dr. Gage started his lab in December 2023 and focuses on understanding how endothelial cells gain and maintain organ specific functions to build new therapies for human liver diseases where these functions are lost. The Gage lab’s primary research model is human pluripotent stem cells which are differentiated to become endothelial cells that can be transplanted in mice where they durably engraft and become functional. This research model and approach has potential therapeutic value as delivery of stem cell-derived endothelial cells to mouse models of Hemophilia A resulted in correction of the severe bleeding disorder by sustained production of bioactive coagulation factors. Building on this cell-based therapy leverages many computational, genetic, and molecular approaches to identify and apply new therapies aimed at controlling endothelial function to fight disease.
Prof. Edward Tuddenham
Emeritus Professor of Haemophilia
University College London
Gene therapy for haemophilia B widely adopted but problems remain for haemophilia A
Professor Edward (Ted) Tuddenham's journey into haemophilia began in 1969 at the Royal Victoria Infirmary in Liverpool, where he treated haemophilic patients. His interest deepened in Cardiff under Arthur Bloom, where he investigated the relationship between Von Willebrand Disease and Haemophilia A. Tuddenham dedicated seven years to purifying factor VIII, first at the University of Connecticut and then at the Royal Free Hospital Haemophilia Centre. His work led to the complete purification of factor VIII, enabling its amino acid sequencing and the cloning of its gene. These breakthroughs facilitated accurate carrier testing, antenatal diagnosis, and the discovery of mutations causing haemophilia A.
In 1987, Tuddenham established the Haemostasis Research Group for the Medical Research Council, focusing on rare bleeding disorders. In 2006, he returned to the Royal Free Hospital to pursue gene therapy for haemophilia. His team demonstrated the safety and efficacy of gene therapy for haemophilia B, using a vector that allowed high levels of factor IX expression. This resulted in most patients discontinuing replacement therapy and remaining free of spontaneous bleeding for up to 13 years. This vector was later modified by Uniqure, leading to the marketing approval of Hemgenix in 2023.
Collaborating with Amit Nathwani's laboratory, Tuddenham also helped develop a vector for transferring the factor VIII gene to treat Haemophilia A, culminating in market approval in the EU in 2022. He continues his work on gene therapy trials for haemophilia and other rare bleeding disorders, with 350 publications, an H-index of 74, and over 21,000 citations.
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Join us for an interactive workshop and Q&A led by global CGT recruiters who will provide insights on how to land your dream job and craft a path to elevate your career.
Presented by the ISCT ESP Mentoring Working Group
March 11, 2024
Webinar Description:
Join us for an interactive workshop and Q&A led by global CGT recruiters who will provide insights on how to land your dream job and craft a path to elevate your career.
Jen Metivier, MS
Associate Director, Talent Acquisition
Obsidian Therapeutics
Emily Thurley
Principal Consultant, Tech Ops & Quality
Skills Alliance
Alice Sandall
Consultant, Tech Ops & Quality
Skills Alliance
Maurice Thorton
Managing Director
Headcount Recruitment
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Contains 3 Component(s)
This webinar is an informed discussion around the current realities and challenges of cell and gene therapies for sickle cell disease with panelists from various roles/viewpoints in the field.
Presented by the ISCT ESP Mentoring Program
April 11, 2024
Webinar Description:
This webinar is an informed discussion around the current realities and challenges of cell and gene therapies for sickle cell disease with panelists from various roles/viewpoints in the field.
Kevin Bosse, PhD, RAC-US
Director of Research Regulatory Affairs
Nationwide Children's Hospital, United States
Director of the Office of Research Regulatory Affairs at Nationwide Children’s Hospital. As a RAC-certified regulatory professional, he advises the development and regulatory strategy of novel drugs, biologics and devices with extensive focus on cell and gene therapy.
Ms Victoria Gray
First sickle cell disease patient, who volunteered in 2019 for the exa-cel therapy trial, sponsored by Vertex Pharmaceuticals and CRISPR Therapeutics. Mother of four from Forest, Mississippi, she is a sickle cell warrior having received a CRISPR-based therapy for SCD.
Ms Michele Salter
Chair of the Sickle Cell Society (UK). Michele was formerly the Vice-Chair & Treasurer and has been working for the Sickle Cell Society since 2014, contributing to its goal to support and represent people affected by sickle cell, to improve their overall quality of life.
Akshay Sharma, MD
Clinician-Scientist
Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children’s Research Hospital
Clinician-scientist in the Department of Bone Marrow Transplantation and Cellular Therapy at St. Jude Children’s Research Hospital. He is an expert in development of novel gene editing therapeutics, and his research includes transplant and gene therapy clinical trials for patients with SCD.
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Contains 2 Component(s)
Join us for an engaging webinar where we explore the art of crafting and delivering impactful elevator pitch presentations. Discover proven strategies and techniques to captivate your audience and leave a lasting impression when showcasing your innovative research. Our speakers, including previous elevator pitch judges and winners, will share invaluable insights on composing your pitch, refining your message, and mastering delivery techniques to confidently showcase your ideas. Plus, you'll have dedicated exercise time to practice and receive feedback on your pitch. Whether you're a seasoned professional or just starting out, this webinar will equip you with the skills and confidence to make every elevator pitch count. Don't miss out – register now and elevate your presentation game!
Co-organized by Cytotherapy and the ESP Committee Community Engagement Working Group
May 16, 2024
Webinar Description:
Join us for an engaging webinar where we explore the art of crafting and delivering impactful elevator pitch presentations. Discover proven strategies and techniques to captivate your audience and leave a lasting impression when showcasing your innovative research. Our speakers, including previous elevator pitch judges and winners, will share invaluable insights on composing your pitch, refining your message, and mastering delivery techniques to confidently showcase your ideas. Plus, you'll have dedicated exercise time to practice and receive feedback on your pitch. Whether you're a seasoned professional or just starting out, this webinar will equip you with the skills and confidence to make every elevator pitch count. Don't miss out – register now and elevate your presentation game!
Key Learning Objectives:
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To develop and demonstrate effective skills in delivering elevator pitch presentations.
Daniel J. Weiss, MD, PhD (Moderator)
Professor, ISCT 2024 Co-Chair
University of Vermont, US
Nikita Patel, MSci, PhD (Moderator)
Senior Scientist
INmune Bio
Rachel Burga, PhD (Moderator)
Principal Scientist, Early Stage Professionals Committee (ESP) Co-Chair
Obsidian Therapeutics
Kathryn Strange, MSc, PhD
Postdoctoral Research Associate
The Francis Crick Institute, Kings College London
Elani Wiest, PhD
Process Development Associate Staff Scientist
Mayo Clinic
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