The HESI Cell Therapy – TRAcking, Circulation, & Safety (CT-TRACS) Committee will present a webinar on Tuesday, 28 September 2021 featuring three speakers, included in the program details below, who will share regulatory perspectives from both sides of the Atlantic. This activity is part of the CT-TRACS Committee’s efforts to continue filling the knowledge gap about methods that can assess the fate of therapeutic cells and/or can help the timely identification of eventual unexpected effects while highlighting the regulatory component of these endeavors.
This webinar is co-organized by EATRIS and the HESI CT-TRACS Committee and will be open to the public. Participants in the audience will have the opportunity to ask questions through the webinar platform.
Medicines Evaluation Board, The Netherlands
Tineke van den Hoorn is a non-clinical pharmacology, pharmacokinetics and toxicology assessor at the MEB (Medicines Evaluation Board) and also participates as such in EMA (European Medicines Agency) regulatory activities. In addition, she is co-chair of the HESI (Health and Environmental Sciences Institute) committee CT-TRACS (Cell Therapy – TRAcking, Circulation, & Safety), since 2019. Her work and interests focus on advanced therapies and immunological products. Before joining the MEB she held a scientist position at TNO and a position as scientific writer at Genmab B.V. She holds a Master’s degree in Biology with a focus on cell biology and structural biology from Utrecht University and she earned a PhD in tumor immunology at Leiden University with Prof. Jacques Neefjes.
US Food and Drug Administration (FDA), CBER/OTAT, USA
Kate Dabirsiaghi is a pharmacology and toxicology reviewer in the Office of Tissues and Advanced Therapies at the United States FDA. She received her doctorate of veterinary medicine from the University of Pennsylvania, with a focus on small animal clinical medicine. Prior to joining FDA, she was a practicing veterinarian and medical director of a large veterinary hospital. She holds a Master’s degree in molecular genetics and a Master’s degree in cellular biology, developmental biology, and biophysics from Johns Hopkins University.
Marta has a Pharmacy degree and a Specialist postgraduate degree qualification in Industrial Pharmacy and Galenic Formulation. She has over seven years’ experience in drug development in the pharma Industry as R&D Project Manager on the development of drug candidates, and over six years’ experience in regulatory affairs. As Scientific and Regulatory Affairs Director at Asphalion, Marta is responsible of scientific and medical writing of regulatory documents working in regulatory strategy, regulatory roadmaps, ATMP designation, scientific advice and qualification of novel methodologies for biologicals, biomaterials, medical devices, and advanced therapies. She also manages regulatory Work Packages of H2020 projects.
Passionate about delivering Advanced Therapeutic Medicinal Products (ATMPs) to patients. Focused on developing technology that will enable successful translation and commercialization.
Biological Scientist, with a background in inflammation, biology of ageing and connective tissues. Applying this research in the fields of auto-immune, degenerative diseases and oncology. Experience gained in academia at the Universities of Cambridge and Newcastle-upon-Tyne, combined with over 15 years of industrial research with Unilever R&D, GE Healthcare Life Sciences and now Cytiva. Recent roles have been supporting the Cell Therapy Industry through and the application of in-vivo imaging to enable safety, efficacy and MoA studies, and development of tools and technology for cell therapy manufacturing and cryo-preservation. Current role is focused on Leading a global team of biologists and engineers developing technology to enable the development of equipment, consumables, software and processes for the manufacture of cell and gene therapies.
Introduction: Background (HESI & CT-TRACS) and Setting the Stage (Bill Shingleton, Cytiva, CT-TRACS Committee Co-Chair)
Talk 1: Regulatory Considerations for Preclinical Development of Cell-Based Therapies – Learnings for the Future (Tineke van den Hoorn, Medicines Evaluation Board, The Netherlands)
Regulatory guidelines on preclinical development of cell-based products should not be used as a tick-box but mostly provide lines for thought for design of the development program. Cell-based products are a diverse group of products, therefore a one-size-fits-all guideline is not possible. Comparison of the development programs of cell-based products (from scientific advise procedures) that are categorized based on their cell origin (autologous versus allogeneic) and on the genetic manipulation status reveals quite some variability. A comparison of the development programs of similar type of cell-based products (from registration procedures) seems to provide a more specific framework of relevant studies.
Talk 2: Preclinical Considerations for Cellular Therapies – A CBER/FDA Approach (Kate Dabirsiaghi, US Food and Drug Administration, CBER/OTAT, USA)
In this talk, Dr. Dabirsiaghi will describe the organizational structure of the Office of Tissues and Advanced Therapies in the Center for Biologics Evaluation and Research at FDA (FDA/CBER/OTAT) and the products they regulate. She will discuss preclinical considerations for assessing the safety and distribution of cell-based products as well as principles for selecting appropriate animal models for these studies. Last, Dr. Dabirsiaghi will describe opportunities for early interaction with CBER/OTAT.
Talk 3: Case Study, nTRACK Project – Utilizing Nanoparticles for Cell Tracking; Lessons Learned From the Regulatory Process in the EU (Marta Rayo Lunar, Asphalion, Spain)
n-TRACK is a H2020 EU funded project developing nanoparticles for tracking of stem cell therapy on muscle regeneration. As it is not straightforward to define the regulatory pathway to develop a product such as n-TRACK, in this talk Marta Rayo Lunar will present this case-study focusing on the knowledge gained on regulatory aspects and preclinical development after several regulatory actions carried out in the EU.
Presentations will be followed by a Q&A/panel discussion with all speakers (~20-30 minutes), moderated by Bill Shingleton, Cytiva, CT-TRACS Committee Co-Chair.