ADVANCED THERAPIES XCHANGE EUROPE
Brussels, Belgium
June 26, 2023
Welcome to hubXchange’s Advanced Therapies Xchange Europe 2023, bringing together executives from pharma and biotech to address and find solutions to the key issues faced in cell and gene therapeutics.
Discussion topics will cover Cell Tx Development, Gene Tx Development, C&G Tx Bioprocessing and C&G Tx Manufacturing.
Take advantage of this unique highly interactive meeting format designed for maximum engagement, collaboration and networking with your peers.
Venue: DoubleTree by Hilton Brussels City, Ginestestraat 3 Rue Gineste, 1210 Brussels, Belgium
SNAPSHOTS OF DISCUSSION TOPICS
- Enhancing CAR-T cell efficacy
- Alternatives to autologous-allogenic and in vivo cell therapies
- The promise and challenges of PSC-CAR-NK cell therapy
- Challenges and future development of the CNS gene therapies
- Overcoming challenges posed by the immune system to gene transfer
- Demonstrating efficacy and value for one-and-done therapies
- Strategies to implement flexibility in continuous biomanufacturing
- Optimizing downstream purification
- Modelling and simulations as a tool to scale-up processes
- Tackling the challenges of AAV vector manufacturing
- Adapting analytics and quality control in cell therapy manufacturing to ensure adequate safety, purity and potency of final products
- Future of autologous cell therapy
Full Xchange Agenda
Click on each track for detailed agenda
Cell Tx Development
Implementing NGS and predictive analytics in cell therapy development
- What are the barriers to routine implementation of NGS during cell therapy development?
- How are people using NGS data in decision making, and at what stages of product development?
- What predictive analytics are being implemented to integrate internal and external data?
Director, Systems Biology, Notch Therapeutics
Dan Kirouac leads the Systems Biology department at Notch Therapeutics, integrating bioinformatics, dynamical systems modelling and machine learning to design and deliver the next generation of T cell therapies. Prior to joining Notch in 2020, he held scientific positions in large pharma (Genentech), mid-size biotech (Merrimack Pharmaceuticals) and consulting (Applied BioMath), and has been developing mathematical models of biological systems for almost 20 years. Dan did post-doctoral training at MIT and Harvard Medical School, holds a PhD in Biomedical Engineering from the University of Toronto, and Bachelors’ in both Chemical Engineering and Genetics from the University of Western Ontario.
Networking Lunch
Spotlight Presentation
Outsource the compliance, control the manufacturing: A different way of thinking about biomanufacturing
- Understand biomanufacturing landscape and traditional options available for clinical and commercial manufacturing
- Provide data and insights on how the industry utilizes those traditional options
- Introduce a hybrid option that minimizes compliance and capital risk while maximizing flexibility in development and production
Vice President of Client Development, Azzur Group
David Frank, VP of Client Development, leads customer-facing initiatives for Azzur Group nationwide including Azzur Cleanrooms on Demand™, consulting and advisory services, labs, and training. For the past decade, David has consulted some of the world’s largest pharmaceuticals, medical devices, technology, and financial services companies, aligning people, processes, and technology to drive sustainable business results. He has been recognized for leading award-winning teams over the past 20 years and is passionate about developing individuals into leaders. David received his Bachelor of Arts in Economics from The Pennsylvania State University and is a Lean Six Sigma Master Black Belt and PMP.
14:50 – 15:20
Poster Session
Optimizing T Cell Therapy Outcomes with TCR Engineering: A Path to Success in Cancer Treatment
T cell therapies have shown promising therapeutic potential in a variety of contexts, from in vitro studies to advanced clinical trials. However, the high cost and complexity of such
therapies can significantly slow down their technological development.
Our interdisciplinary team has developed a suite of computational solutions to streamline the end-to-end development of cellular therapies for cancer treatment. These services can help identify T-cell targets, predict epitope presentation, describe the dynamics of T-cell
repertoires and tumor microenvironment through NGS data analysis, and identify potential T-cell off-target toxicities. The TCR optimization module also enables us to engineer T-cell receptors (TCRs) to enhance the therapeutic potential of T-cell candidates by proposing mutations designed to lengthen the interaction time between TCRs and their targets. Such services can be extended to other therapeutic modalities targeting antigens (CAR-Ts, NKs, antibodies, nanobodies, DARPins, etc.).
Lead Scientist, Ardigen
Giovanni Mazzocco is a computational biologist expert with over a decade of experience in both academic and commercial projects. He has collaborated with a variety of institutions, including universities, scientific institutes, and medical research centers, as well as pharmaceutical and biotechnology companies. Throughout his career, Giovanni hasconducted research in various areas of bioinformatics and computational biology, including enzyme discovery, molecular modeling, protein dynamics and interactions, chromatin modeling, design of statistical learning methods for predicting protein interactions, vaccine design, NGS analysis and more.
Giovanni is currently working at Ardigen, where he is using AI to advance precision medicine in oncology. He is leading the TCR optimization project in the immunology team, and is responsible for proposing and exploring new scientific directions for the Immunology Business Unit.
Maximizing clinical benefit in solid tumor therapies: Combination therapies and other strategies
- Prevention of exhaustion: Checkpoints (PD-1 and Combos), Epigenetic Modulators and others (chemo)
- Prevention of and overcoming resistance: Drug sequencing, multiple targets, combinations
- Expanding the addressable market: Minimizing toxicity, minimizing in-patient time, cost
Chief Operations Officer, VisiCELL Medical
George Smith is the COO at VisiCELL Medical, developing cell therapy imaging diagnostics, and is an advisor to advanced therapy companies. Recently, he led strategy and development of next generation TIL therapies at Myst/Turnstone Biologics. He founded the IQVIA Cell and Gene Therapy Center, helping companies develop advanced therapies. He is a SME for clinical and preclinical development strategy, pharmacology, translational medicine and asset evaluation. He has worked for small and large biopharma companies for a wide variety of therapeutic areas and modalities from concept through Phase III, including inventing Paxlovid. Dr. Smith received his Ph.D. and MBA from UCSD.
16:30 – 17:30
Overcoming resistance to currently approved autologous engineered T cell based therapies
- ~80% ORRR, 50% CR yet in 3rd line, >60% of patients relapse from currently approved CAR T Therapy across indications
- Advanced and next generation cell therapy product development needed to meet emerging CAR refractory patient population
- Solid tumor indications remain and unmet need
Vice President, Translational Medicine, Cargo Therapeutics
Gene Tx Development
Challenges to effective in vivo gene therapy with CRISPR/Cas
- What are the new CRISPR systems for versatile gene editing?
- How to increase efficiency of payload delivery in vivo?
- How to achieve cell-specific targeting?
- What is the best way to evaluate safety and reduce risk of off-target editing?
Director, Vaxart
William Sun is currently Director of Molecular Virology at Vaxart Inc. He is an experienced molecular and cell biologist with broad scientific background. He has led research teams engaged in drug discovery and stem cell therapy, in areas of metabolic diseases, cardiology, oncology, infectious diseases, and regenerative medicine. He was previously Principal Research Scientist at the Institute of Bioengineering and Nanotechnology, and Group Leader at the Experimental Therapeutics Centre, A*STAR. His technical expertise includes molecular cloning, assay development, recombinant antibody technology, human pluripotent stem cell culture and differentiation, and human genome engineering.
Networking Lunch
Spotlight Presentation
Outsource the compliance, control the manufacturing: A different way of thinking about biomanufacturing
- Understand biomanufacturing landscape and traditional options available for clinical and commercial manufacturing
- Provide data and insights on how the industry utilizes those traditional options
- Introduce a hybrid option that minimizes compliance and capital risk while maximizing flexibility in development and production
Vice President of Client Development, Azzur Group
David Frank, VP of Client Development, leads customer-facing initiatives for Azzur Group nationwide including Azzur Cleanrooms on Demand™, consulting and advisory services, labs, and training. For the past decade, David has consulted some of the world’s largest pharmaceuticals, medical devices, technology, and financial services companies, aligning people, processes, and technology to drive sustainable business results. He has been recognized for leading award-winning teams over the past 20 years and is passionate about developing individuals into leaders. David received his Bachelor of Arts in Economics from The Pennsylvania State University and is a Lean Six Sigma Master Black Belt and PMP.
14:50 – 15:20
Poster Session
Optimizing T Cell Therapy Outcomes with TCR Engineering: A Path to Success in Cancer Treatment
T cell therapies have shown promising therapeutic potential in a variety of contexts, from in vitro studies to advanced clinical trials. However, the high cost and complexity of such
therapies can significantly slow down their technological development.
Our interdisciplinary team has developed a suite of computational solutions to streamline the end-to-end development of cellular therapies for cancer treatment. These services can help identify T-cell targets, predict epitope presentation, describe the dynamics of T-cell
repertoires and tumor microenvironment through NGS data analysis, and identify potential T-cell off-target toxicities. The TCR optimization module also enables us to engineer T-cell receptors (TCRs) to enhance the therapeutic potential of T-cell candidates by proposing mutations designed to lengthen the interaction time between TCRs and their targets. Such services can be extended to other therapeutic modalities targeting antigens (CAR-Ts, NKs, antibodies, nanobodies, DARPins, etc.).
Lead Scientist, Ardigen
Giovanni Mazzocco is a computational biologist expert with over a decade of experience in both academic and commercial projects. He has collaborated with a variety of institutions, including universities, scientific institutes, and medical research centers, as well as pharmaceutical and biotechnology companies. Throughout his career, Giovanni hasconducted research in various areas of bioinformatics and computational biology, including
enzyme discovery, molecular modeling, protein dynamics and interactions, chromatin modeling, design of statistical learning methods for predicting protein interactions, vaccine design, NGS analysis and more.
Giovanni is currently working at Ardigen, where he is using AI to advance precision medicine in oncology. He is leading the TCR optimization project in the immunology team, and is
responsible for proposing and exploring new scientific directions for the Immunology Business Unit.
Demonstrating efficacy and value for one-and-done therapies
- Key considerations for defining inclusion and exclusion criteria
- Discuss options for a well-controlled trial in a small, variable population with minimal natural history data
- Challenges in establishing meaningful clinical endpoints early in development to establish durability
Director of Gene Therapy Program Management, Astellas Gene Therapies
Jill Woloszynek is a Global Development Project Lead at Astellas Gene Therapies where she leads global program strategy from the research stage through life cycle management. She is focused on utilizing gene therapy as a method to treat rare diseases. For the last 9 years, Jill has focused on gene therapy in many capacities; non-clinical research, gene therapy program development, clinical development, and competitive intelligence. She has played key roles on several clinical stage gene therapy programs.
C&G Tx Bioprocessing
Optimizing downstream purification
- Reduction of empty capsid in bioprocessing of AAV vectors
- Reduction of HCP and DNA to safe levels over the overall concentration fold required for Bulk Drug Substance
- Reduction of AAV particles containing DNA fragments derived from host cell DNA, helper or plasmid DNA
- New technologies needed for novel capsids
Associate Director, CMC Lead, Astellas Gene Therapies
Damiano has worked in the biopharmaceutical space for the last fifteen years in both industry and academia. He graduated with a PhD in Biochemical Engineering from University College London while focusing on optimization of downstream process for a therapeutic enzyme intended to treat rare disease POMPE. He has worked at Biogen in analytical development and more recently at Genentech and Tenaya Therapeutics as global regulatory project manager. After a two year Cell and Gene therapy CMC Regulatory consulting experience, Damiano has joined the Astellas Gene therapy team where he currently serves as Associate Director of CMC Lead and Project management for multiple pre/clinical stage programs.
Spotlight Presentation
Outsource the compliance, control the manufacturing: A different way of thinking about biomanufacturing
- Understand biomanufacturing landscape and traditional options available for clinical and commercial manufacturing
- Provide data and insights on how the industry utilizes those traditional options
- Introduce a hybrid option that minimizes compliance and capital risk while maximizing flexibility in development and production
Vice President of Client Development, Azzur Group
David Frank, VP of Client Development, leads customer-facing initiatives for Azzur Group nationwide including Azzur Cleanrooms on Demand™, consulting and advisory services, labs, and training. For the past decade, David has consulted some of the world’s largest pharmaceuticals, medical devices, technology, and financial services companies, aligning people, processes, and technology to drive sustainable business results. He has been recognized for leading award-winning teams over the past 20 years and is passionate about developing individuals into leaders. David received his Bachelor of Arts in Economics from The Pennsylvania State University and is a Lean Six Sigma Master Black Belt and PMP.
14:50 – 15:20
Poster Session
Optimizing T Cell Therapy Outcomes with TCR Engineering: A Path to Success in Cancer Treatment
T cell therapies have shown promising therapeutic potential in a variety of contexts, from in vitro studies to advanced clinical trials. However, the high cost and complexity of such
therapies can significantly slow down their technological development.
Our interdisciplinary team has developed a suite of computational solutions to streamline the end-to-end development of cellular therapies for cancer treatment. These services can help identify T-cell targets, predict epitope presentation, describe the dynamics of T-cell
repertoires and tumor microenvironment through NGS data analysis, and identify potential T-cell off-target toxicities. The TCR optimization module also enables us to engineer T-cell receptors (TCRs) to enhance the therapeutic potential of T-cell candidates by proposing mutations designed to lengthen the interaction time between TCRs and their targets. Such services can be extended to other therapeutic modalities targeting antigens (CAR-Ts, NKs, antibodies, nanobodies, DARPins, etc.).
Lead Scientist, Ardigen
Giovanni Mazzocco is a computational biologist expert with over a decade of experience in both academic and commercial projects. He has collaborated with a variety of institutions, including universities, scientific institutes, and medical research centers, as well as pharmaceutical and biotechnology companies. Throughout his career, Giovanni hasconducted research in various areas of bioinformatics and computational biology, including enzyme discovery, molecular modeling, protein dynamics and interactions, chromatin modeling, design of statistical learning methods for predicting protein interactions, vaccine design, NGS analysis and more.
Giovanni is currently working at Ardigen, where he is using AI to advance precision medicine in oncology. He is leading the TCR optimization project in the immunology team, and is responsible for proposing and exploring new scientific directions for the Immunology Business Unit.
16:30 – 17:30
- Viral vs non-viral
- Cold chain monitoring for Harvest/Fill/Finish
- Cryoformulation
Vice President, Head of Tech Ops, Senti Biosciences
Dr. Giedlin is currently the VP, Head of Tech Ops at Senti BioSciences supporting the allogeneic NK-CAR programs. Before his joining Senti, he led the PD groups at PACT Pharma (non-viral process for generating autologous neoTcR+ T cells), Poseida (non-viral process for generating autologous CAR-T’s), and Novartis (clinical and commercial manufacturing process for Kymriah® ). His experience covers over 25 years of various approaches to harness the immune response to treat cancer and infectious disease, starting with Proleukin® rIL-2 (aldesleukin; Chiron), Oncolytic adenovirus (Onyx), Listeria-based cancer vaccines (Cerus), and CCR5 knockout CD4+ T cells for HIV+ patients (Sangamo). In addition, he led the implementation of AAV clinical manufacturing to support the Hemophilia, LSD, and neurodegenerative disease programs.
C&G Tx Manufacturing
- Regional centers versus onsite/academic hospital manufacturing
- Certificate of analysis and release considerations
- Regulatory issues and oversight of CQAs and CPPs
Chief Cellular Therapy Officer, Nurix Therapeutics
Michael Lotze has served as Chief Cellular Therapy Officer of Nurix since June 2020. He is a leading clinician scientist with more than 30 years of experience in immunology and clinical medicine, dedicating his efforts to the advancement of translational research, particularly in immunotherapy for cancer including dendritic cell, T cell, and cytokine therapies.
He is the co-inventor of multiple patents in dendritic cell vaccines and antigen discovery, and tumor infiltrating lymphocyte therapy. He previously held leadership roles at Iovance Biotherapeutics and GlaxoSmithKline. Prior to joining Nurix, Michael served as professor of surgery, immunology, and bioengineering, vice chair of research at the University of Pittsburgh Medical Center Hillman Cancer Center.
Over the course of his career, he has authored more than 500 publications and several books. Michael holds an M.D. from Northwestern University and completed his postdoctoral training at the National Cancer Institute.
Defining gene-editing nucleases and plasmid DNA manufacturing strategies
- Optimization & Development: Invest upfront to ensure downstream manufacturing efficiencies. Defining the balance between speed & time investment
- Facility & Quality Requirements: Understanding your CMO’s facility infrastructure & quality systems to ensure suitability from toxicology through to late-clinical and commercial
- Analytics & Documentation: Defining appropriate analytics and documentation for phased-appropriate manufacturing to meet regulatory requirements
- Scale-Up: Build a strategy and CMO partnership that bridges to large-scale manufacturing and validation capabilities
Vice President of Commercial, Akron Biotech
Robert Margolin brings 14 years of business leadership and sales experience in the cell and gene therapy market, with a focus on business development and corporate strategy and execution. His experience ranges across cell therapy drug product and ancillary material manufacturing. Robert’s deep knowledge of clinical and commercial operations has driven process development, scale-up, and design and configuration of electronic systems and logistics platforms for each stage of development. Robert has worked closely with the field’s leading companies and industry organizations throughout the U.S. and Europe. Additionally, Robert played an instrumental role in the formation and growth of the Alliance for Regenerative Medicine, serving as its vice president of communications, along with launching several of the field’s preeminent conferences and events. Robert’s network spans the entire industry encompassing KOLs from business, science, medicine, regulatory, policy and advocacy.
Networking Lunch
Spotlight Presentation
Outsource the compliance, control the manufacturing: A different way of thinking about biomanufacturing
- Understand biomanufacturing landscape and traditional options available for clinical and commercial manufacturing
- Provide data and insights on how the industry utilizes those traditional options
- Introduce a hybrid option that minimizes compliance and capital risk while maximizing flexibility in development and production
Vice President of Client Development, Azzur Group
David Frank, VP of Client Development, leads customer-facing initiatives for Azzur Group nationwide including Azzur Cleanrooms on Demand™, consulting and advisory services, labs, and training. For the past decade, David has consulted some of the world’s largest pharmaceuticals, medical devices, technology, and financial services companies, aligning people, processes, and technology to drive sustainable business results. He has been recognized for leading award-winning teams over the past 20 years and is passionate about developing individuals into leaders. David received his Bachelor of Arts in Economics from The Pennsylvania State University and is a Lean Six Sigma Master Black Belt and PMP.
14:50 – 15:20
Poster Session
Optimizing T Cell Therapy Outcomes with TCR Engineering: A Path to Success in Cancer Treatment
T cell therapies have shown promising therapeutic potential in a variety of contexts, from in vitro studies to advanced clinical trials. However, the high cost and complexity of such
therapies can significantly slow down their technological development.
Our interdisciplinary team has developed a suite of computational solutions to streamline the end-to-end development of cellular therapies for cancer treatment. These services can help identify T-cell targets, predict epitope presentation, describe the dynamics of T-cell
repertoires and tumor microenvironment through NGS data analysis, and identify potential T-cell off-target toxicities. The TCR optimization module also enables us to engineer T-cell receptors (TCRs) to enhance the therapeutic potential of T-cell candidates by proposing mutations designed to lengthen the interaction time between TCRs and their targets. Such services can be extended to other therapeutic modalities targeting antigens (CAR-Ts, NKs, antibodies, nanobodies, DARPins, etc.).
Lead Scientist, Ardigen
Giovanni Mazzocco is a computational biologist expert with over a decade of experience in both academic and commercial projects. He has collaborated with a variety of institutions, including universities, scientific institutes, and medical research centers, as well as pharmaceutical and biotechnology companies. Throughout his career, Giovanni hasconducted research in various areas of bioinformatics and computational biology, including enzyme discovery, molecular modeling, protein dynamics and interactions, chromatin modeling, design of statistical learning methods for predicting protein interactions, vaccine design, NGS analysis and more.
Giovanni is currently working at Ardigen, where he is using AI to advance precision medicine in oncology. He is leading the TCR optimization project in the immunology team, and is responsible for proposing and exploring new scientific directions for the Immunology Business Unit.
Which indications are most and least likely to benefit from autologous CT?
What challenges are companies with autologous CT programs currently navigating?
How should a company think about questions of scale and access when developing a new autologous CT?
What solutions exist or need to be developed to make CT more clinically impactful? Is truly personalized medicine sustainable?
What innovations would fundamentally change the autologous CT space? How far away are we from making these real?
Vice President, Supply Chain Management & Business Operations, Sangamo Therapeutics
Heather Erickson serves as Vice President of Supply Chain Management and Business Operations at Sangamo Therapeutics, a publicly traded, clinical-stage company with programs in gene therapy, genome editing, cell therapy, and gene regulation. She joined Sangamo in 2016 as Chief of Staff, a post she held for two years. Previous roles included serving as President and CEO of the Life Sciences Foundation and founding President of MedTech Association. Earlier in her career, she served as Senior Consultant at Edgar Dunn & Company, a boutique strategy consultancy. She currently serves on several industry and community Boards.
16:30 – 17:30
- Challenges in upstream and downstream AAV production
- The need for fit-for-purpose standards
- CMC requirements for potency assays
Principal Scientist, CERo Therapeutics
Edson Oliveira is a Principal Scientist leading the viral vector core of CERo Therapeutics. He is an accomplished molecular virologist and immunologist with 8+ years of post-PhD experience and has authored over 20 publications in the field. Edson is currently dedicated in developing viral vector-based approaches to support cell therapies primarily to treat hematologic tumors. He has experience in viral vector production systems including upstream, downstream and analytics with focus on lentivirus and adeno-associated virus. In a previous role, Edson served Thermo Fisher Scientific contributing to the early launch of a successful AAV production system (Gibco™ AAV-MAX) as well as leading research to develop ELISA-based residual assay to QC viral vector productions. Edson went to Pharmacy School at the Federal University of Rio de Janeiro, holds a PhD in Cell and Molecular Biology from Oswaldo Cruz Foundation (Fiocruz) in Brazil and completed his postdoctoral training at the University of Illinois Chicago (UIC).