Momentum for Cell and Gene Therapy Symposium:
The speakers and their presentations:
Dr. Bruce Levine, Ph.D., Barbara and Edward Netter Professor in Cancer Gene Therapy, is the Founding Director of the Clinical Cell and Vaccine Production Facility (CVPF) in the Department of Pathology and Laboratory Medicine and the Abramson Cancer Center, Perelman School of Medicine, University of Pennsylvania. He received a B.A. (Biology) from Penn and a Ph.D. in Immunology and Infectious Diseases from Johns Hopkins. First-in-human adoptive immunotherapy trials include the first use of a lentiviral vector, the first infusions of gene edited cells, and the first use of lentivirally-modified cells to treat cancer. Dr. Levine is co-inventor of the first FDA approved gene therapy (Kymriah), chimeric antigen receptor T cells for leukemia and lymphoma, licensed to Novartis. Dr. Levine is co-inventor on 31 issued US patents and co-author of >200 manuscripts and book chapters with a Google Scholar citation h-index of 110. He is a Co-Founder of Tmunity Therapeutics, and of Capstan Therapeutics both spinouts of the University of Pennsylvania.
About his presentation:
“From Innovation to Translation to Patients: The Future of Genetically Engineered T-Cells for Human Therapeutics“:
- Gene-modified T cells, especially CAR-T cells targeting CD19, have shown durable complete responses in patients with relapsed or refractory B cell leukemias and lymphomas.
- The development of new genetically modified T cell designs, such as switches and potency enhancements, is crucial for effectively targeting solid tumors.
- Enhancing patient access to advanced cell and gene therapies involves addressing unique challenges in determining dosage, potency, and pharmacokinetics of living drugs, alongside advancements in automation, engineering, and health policy.
Joerg Ahlgrimm is the Chief Executive Officer at SK pharmteco. He is an Operations and Supply Chain Management Executive who has served in multi-billion dollar companies operating in biotech, vaccines, pharmaceutical and medical device markets. He is the former Head of Global Operations Pharma and Biotech at Lonza AG where he was responsible for 37 sites on four continents. Prior to Lonza, he headed the Manufacturing network of Baxter Bioscience/Baxalta with 17 plants, a significant CMO network and the largest plasma collection division of the company. His prior roles also included responsibility for Supply Chain for Baxter Bioscience. Joerg has a passion for saving lives which is his main motivator to work in healthcare. He enjoys leading teams to achieve unprecedented performance levels and to create a work environment where people bring their best. In 2014 he was awarded “Emerging Pharma Leader.”
Marion Alcantara, MD, Ph.D., was trained as a MD in onco-hematology and has a university degree in cell therapy and a PhD in immunology.
Her clinical activity is focused on coordinating autologous stem cell transplant and cell therapy.
She is the medical director of CellActIon (Cell therapy Acceleration and Innovation), a structure from Institut Curie and also part of the Paris Saclay Cancer Cluster ecosystem, aiming at bringing innovation in order to develop effective cell immunotherapies.
About her presentation:
“Improving the efficacy of chimeric antigen receptor CAR-T cells for primary central nervous system lymphoma“:
- CD19 CAR-T therapy induces high response rates for PCNSL patients but most of them relapse;
- We have developed preclinical models of PCNSL and showed that an epigenetic reprogramming of CD19 CAR-T cells may be beneficial in this disease.
Annelise Bennaceur-Griscelli, MD, Ph.D., is full professor of hematology at the Faculty of Medicine of Paris Saclay University. She holds since 2004 the chairperson position at the Division of Cytogenetic and Molecular Onco-Hematology at the Paris Saclay University Hospitals (Paul Brousse, Villejuif France) for precision medicine and target therapy. She is an expert in stem cells and hematopoiesis and a leader in translational research of induced pluripotent stem cells (iPSC) in the field of immuno-oncology. She derived in 2005 the first French human embryonic stem cell lines and led for 20 years (2004-20) the UMRS 935 INSERM “Malignant and Therapeutic Stem Cell Models”. She coordinates since 2012 the National Research Infrastructure INGESTEM and create ‘Center for IPS Cell Therapy’ CiTHERA (https://cithera-ipsc.com) at Genopole (Evry), for clinical compliant iPSC and derivatives and cell and gene therapies. She co-founded the spinoff IPSirius, supported by European Innovation Council ‘Cell and Gene Therapy Challenges’. She is president of the scientific council of the Association ‘Vaincre le Cancer’, member of steering committees of the European Cost-Action ‘Haplo-iPSC’ and the international consortium ‘Global Alliance for iPSC Therapies’, dedicated to the clinical and industrial use of pluripotent stem cells.
About her presentation:
“Novel immunotherapies based on Pluripotent Stem cells: cross immunity against cancer stemness“:
- iPSCs show promise in Immuno-oncology, offering potential as game changers;
- Gene-edited iPSC-derived immune cells developed for versatile adoptive cell and gene therapies;
- Cancer-iPSC-derived organoid models aid in discovering tumor-associated targets for novel CAR therapies, exploiting stem cell properties to enhance immune response against tumor resistance and enabling scalable. manufacturing for a wide range of cancers alongside other immunotherapies.
Dr. Emmanuel Donnadieu, Ph.D, is a group leader at INSERM Institut Cochin in Paris, DR1 CNRS. His work uses fluorescence imaging techniques to monitor T-cell activities in human tumors. His current projects aim at targeting the tumor microenvironment to improve CAR-T cell therapy.
About his presentation:
“Predicting safety and efficacy of engineered T cells using an ex vivo human model“:
- Development of a new human preclinical model to test CAR-T efficacy;
- Identification of obstacles to CAR-T anti-tumor activities;
- Development of strategies to circumvent these obstacles and make CAR-T more effective;
- Establishment of a human model to predict on-target off-tumor toxicities of CAR-T.
Anne Galy, D.Pharm, Ph.D., is the founder and director of the Inserm Accelerator of Technological Research in Genomic Therapy (ART-TG). This laboratory provides a much-needed capacity for academic translational research for gene and cell therapies. ART-TG aims to accelerate the transfer of discoveries towards clinical applications by developing innovative products and their bioproduction processes, in collaboration with public and private partners and in connection with international networks. Anne Galy’s research interest is centered around gene therapy and the immune system. For the past 20 years at Genethon, she has developed hematopoietic stem cell-based gene therapies using lentiviral vectors that have led to several international phase I/II clinical trials in rare primary immune deficiencies or blood disorders. She has also studied unwanted immune responses to viral vectors and developed endogenized vectors with low immunogenicity. More recently she has focused her interest on genetic immunotherapies such as genetically-engineered B cells or CAR-T cells. Author of 125+ articles, and inventor of several patents Anne Galy also has background experience in private biotechnology and academic institutions in the US. She was recently awarded the 2023 Grand Prize of the French national Academy of Pharmacy.
About her presentation:
“Use of CAR-T cells in non-oncology applications. Improving gene therapy by reducing fibrosis“:
- Fibrosis is a pathogenic condition with excessive accumulation of extracellular matrix which is observed in many conditions. For instance, muscle fibrosis occurs in severe muscular dystrophies and is associated with the expression of high levels of the FAP protein;
- FAP-specific CAR-T cells can reduce the markers of muscle fibrosis and enhance the efficacy of gene therapy in a mouse model of Duchenne muscular dystrophy;
- Combining gene therapy with immunotherapy may provide novel therapeutic options to treat diseases in which fibrosis occurs.
Audrey Greenberg is the Chief Marketing Officer of SK pharmteco. Audrey’s area of expertise is in capital structure, business formation, acquisitions, strategic planning and tactical execution. Audrey’s deep industry experience includes biotechnology, real estate, pharma services, and financial services. Prior to being the Chief Marketing Officer for SK pharmteco, Audrey co-founded The Center for Breakthrough Medicines, acquired by SK pharmteco in 2023. Before that, Audrey spent several years in investment banking, public accounting and private equity having worked at some of the largest asset managers in the world with AUM exceeding $100 billion. Audrey is passionate about supporting her community and serves on several non-profit boards including the King of Prussia Business District and is currently an Emissary for Select Greater Philadelphia, a division of the Philadelphia Chamber of Commerce responsible for attracting business to Greater Philadelphia. Audrey was recently selected by Philadelphia Magazine as one of the top trailblazers in Biotech and was featured on the cover of Main Line Today’s Power Women edition. Audrey has an MBA from The Wharton School and is a registered CPA in Illinois.
Dr. John Lee, Ph.D., is an accomplished CAR-T immunobiologist with over 20 years of cancer cell biology expertise spanning small and large molecules, as well as cell-based therapeutics. He possesses more than 15 years of clinical research and cell therapy experience including positions at The Wistar Institute, Janssen Pharmaceuticals, and GlaxoSmithKline. Dr. Lee previously built the Cell Therapy Platform team at Janssen Pharmaceuticals. The group led and supported end-to-end therapy discovery and development across nearly a dozen CAR-T programs, including the recently approved cilta-cel (Carvykti). During his subsequent tenure as Vice President, Head of Oncology Cell Therapy at GlaxoSmithKline, Dr. Lee’s team operated within a heavily matrixed organization to support an array of activities establishing multiple preclinical assets and several clinical-stage lete-cel programs targeting the NY-ESO cancer-testis antigen. Dr. Lee holds a Ph.D from the Brody School of Medicine, an MBA from Penn State University, and a Bachelors of Science from Indiana University of Pennsylvania.
About his presentation:
“CAR-T: Crafting the Future of Medicine, One Cell at a Time“
Dr Philippe Menasché, MD, is a clinical cardiac surgeon at the Hôpital Européen Georges Pompidou, Professor of Thoracic and Cardiovascular Surgery at the University of Paris and responsible for an Inserm team (UMR 970-Paris Cardiovascular Research Center). He also has a part-time affiliation with the Department of Biomedical Engineering of the University of Alabama in Birmingham. The group has a long-standing interest in stem cells for the treatment of heart failure. After the successful completion of a phase I trial testing cardiac progenitors derived from human embryonic stem cells (ESC) embedded in a patch, the recognition of the predominant role of paracrine signaling has led to shift towards an a-cellular cell therapy based on the exclusive use of the secretome to further streamline the clinical translatability of this myocardial repair strategy. A clinical trial testing this approach is currently underway.
About his presentation:
“Extracellular Vesicles for Cardiac Repair:A Translational Experience“
Nawal Ouzren joined Sensorion as CEO in April 2017. She has around 20 years of experience in operational and strategic management within the pharmaceutical and biotech industry. Mrs. Ouzren started out at GE before moving to Baxter, where she was Strategy and Operational Excellence Manager, Quality Operations Director and Senior Director Strategy before becoming Vice President of the BioSimilars business unit. In 2014, she became Vice President of the Global Hemophilia Franchise at Baxalta, which had been incorporated within the Shire group. In 2016, she headed up the Global Genetic Diseases Franchise at Shire, where she supervised all marketing, business and strategy aspects of this global division’s product portfolio. She is a board member of Sensorion and recently was appointed board member of Croda International Plc.
About her presentation:
“Hearing Disorders: a new field for Gene Therapy”
- Intro to Sensorion & its ecosystem;
- Understanding of Hearing Loss (current landscape, causes);
- Application of Gene Therapy to congenital hearing disorders;
- Challenges;
- Senso’s innovative multimodality approach.
Jean-Luc Perfettini, Ph.D., is Director of Research at the National Institute of Health and Medical Research (INSERM), co-director of the research unit INSERM UMR 1030 “Molecular Radiotherapy and Therapeutic Innovation”, Director of the “Cell Death, Immunity and Therapeutic Innovation” team at Gustave Roussy. Jean-Luc Perfettini earned his PhD in the fields of immunology, microbiology and cellular biology at the Pasteur Institute. He obtained his “Advanced Immunology” diploma at the Pasteur Institute in 1998 and then his PhD in Immunology from University of Paris Diderot in 2002. From 2002 to 2006, Jean-Luc Perfettini received a post-doctoral training at Gustave Roussy where he studied cell death modalities and antitumor immunity. Jean-Luc Perfettini became a senior scientist at INSERM in 2006 and Director of Research in 2013. Jean-Luc Perfettini has developed an internationally recognized expertise in the cell death processes, innate immunity and therapeutic innovation in oncology, and is author of more than 83 publications (including articles in Nat Immunol, Nat Med, Nat Cell Biol, Nat Commun and J Exp Med). He has also filed 14 patents related to predictive biomarkers in cancer, nano-medicine and myeloid cell therapies. Jean-Luc Perfettini is coordinating an emerging medical scientific program in innovative biotherapeutics at Gustave Roussy.
About his presentation:
“Next-generation phagocytosis-guided myeloid cell therapy for cancer treatment“:
- Identification of p21-engineered monocyte-based cellular therapy as novel cancer immunotherapy;
- Characterization of tumor homing and in situ differentiation of adoptively transferred p21-engineered monocytes into phagocytosis proficient tumor-associated macrophages;
- in-vivo characterization of antitumoral and immunological activities of adoptively transferred p21-engineered monocytes.
David Sourdive, Ph.D., is a co-founder of Cellectis and joined the Board of Directors in 2000. Dr. Sourdive combines a strong scientific expertise with experience in managing industrial programs bringing innovative technologies to industrial fruition. He served as Executive Vice President, Corporate Development, from 2008 to 2016 and as Executive Vice President, Technical Operations until July, 2019. In addition to his role at Cellectis, Dr. Sourdive has also served on the board of directors of the Mediterranean Institute for Life Sciences. David Sourdive graduated from École Polytechnique, received his Ph.D. in molecular virology at Institut Pasteur and completed a research fellowship in the Emory University Department of Microbiology and Immunology. His management training is from the HEC (Challenge +) and his decade-long experience in industrial program management was acquired at the French Department of Defense (DGA) prior to Cellectis’ inception.
About his presentation:
“Cellectis’ engineer’s approaches: from inventing allogeneic CAR-T products to advanced smart cells“:
- Transforming cell therapy by developing and leveraging the safest and most powerful gene editing approaches;
- Transitioning cell therapy from autologous grafts to robust off-the-shelf allogeneic pharmaceutical products;
- The next step: unlocking the full power of cell therapies by engineering biological scenarios with genomic design of “smart cells”.
Els Verhoeyen, Ph.D., is a research director at the institute for health and medical research (INSERM) with a dual affiliation: the international center for infectiology research (CIRI) in Lyon (France) and the mediteranean molecular medical research center (C3M) in Nice (France). Her research is focused on the development of lentiviral vectors and gene editing tools for gene therapy. In particular via surface display on these vectors with envelopes from heterologous viruses to allow specific transduction of gene therapy target cells. She has engineered lentiviral vectors that allow efficient transduction of T cells, B cells and hematopoietic stem cells. Later on, in collaboration with C. Buchholz she targeted lentivectors to T cells in order to generate chimeric antigen receptor (CAR) T cells directly in vivo in human blood system mice. More recently, she developed virus like particles carrying CAS9 protein and associated gRNAs as RNA which an improved system for delivery of the gene editing machinery (CRISPR/CAS9) in blood cells and organoids.
She is also part of a CRISPR/CAS9 screening Platform to guide researchers in their project (in Nice) and is also founder of a retroviral vector production platform in Lyon.
She is also the president of the French society for cell and gene therapy society (SFTCG) and is in 2022 elected as board member of the European society (ESCGT).
About her presentation:
“Novel targeted lentiviral vectors for in vivo CAR-T cell generation and ‘nanoblades’ for efficient gene editing in blood stem cells and organoids“:
- CD8 receptor-targeted LVs allow in vivo generation of CD19 CAR -T cells and CD4 CAR-T cells to eliminate B cells and malignant T-cells in a T-cell lymphoma model;
- Nanoblades are novel delivery tools for the CRISPR/Cas9 gene editing machinery allowing high level gene editing in blood cells and organoids without inducing toxicity and low off-target effects.
Prof. Ibrahim Yakoub-Agha, MD, Ph.D., is the current chair of the EBMT practice harmonization and guidelines, past chair of the Chronic Malignancies Working Party (CMWP) of EBMT (2018-2022) and past President of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC) (2012-2016), Prof. Ibrahim Yakoub-Agha has served as Head of the Hematopoietic Cell Transplantation and cellular therapy Unit at Lille University Hospital since 2008.
Professor Yakoub-Agha has contributed to the scientific community through his role as the Editor in Chief of “Current Research in Translational Medicine.”. His research output includes authoring or co-authoring more than 500 scientific articles, with a primary focus on allogeneic hematopoietic cell transplantation and innovative gene and cellular therapy.
About his presentation:
“Enhancing Point-of-Care Production of CAR Cells: Strategies and Challenges“:
- Regulations;
- GMP;
- Semi-automated production;
- Hybrid production (industry/academia).
