Cell and gene therapies (CGTs) have shown great potential in treating a wide range of diseases, including cancer, genetic syndromes, autoimmune diseases, and neurological disorders. The number of CGTs is only increasing: in 2021, there were over 1,200 gene therapies in clinical development worldwide, and the US FDA prediction that they would approve 10-20 new gene therapies each year by 2025 is becoming a reality.
While both cell and gene therapies offer long-term benefits for patients, safety and efficacy must be thoroughly evaluated before widespread clinical use, as with any emerging medical technology. As a result, CGTs almost always require long-term follow-up (LTFU) studies, starting in Phase I clinical trials. In this webinar, a panel of experts will cover key regulatory, operational, medical, and scientific insights when considering LTFU studies in the CGT space.
- Regulatory insights for CGT LTFU guidance: early planning and full scope of possible inclusion of parent trials rolling into LTFU (i.e. master protocol possibilities, and more)
- Medical and scientific considerations: ensuring clients really consider the 'need to have' versus 'nice to have' assessments within their schedule of events, which could impact enrollment, retention, and compliance over time
- Operational considerations to increase patient retention
- Engagement with RA entities to comply, and how implications of data can help improve methods of data collection
- Understanding the patient journey and the support that healthcare professionals, researchers, scientists, and industry experts need to provide to meet regulatory requirements
Keith Wonnacott
Vice President Regulatory Affairs at Lexeo Therapeutics
Keith Wonnacott, PhD, joined LEXEO Therapeutics as the Vice President of Regulatory Affairs in November 2021. Prior to joining LEXEO, Dr Wonnacott was an Executive Director of Regulatory Affairs at Pfizer, where he had responsibility for gene therapies within the Rare Disease Business Unit. He provided guidance on regulatory strategy and led regulatory policy for the gene therapy portfolio, which included advancing three late-stage programs into phase 3 clinical trials. Dr Wonnacott also worked at Novartis, where he was a Director of Regulatory CMC and led the development of Module 3 for the Kymriah BLA, which became the first gene therapy product to receive FDA approval in 2017. Dr Wonnacott also held roles of increasing responsibility at the FDA at the start of his career. He spent 13 years in the Division of Cell and Gene Therapies at the FDA, the last 10 of which were as Chief of the Cellular Therapies Branch. In total, Dr Wonnacott has spent his entire career of 2 decades working in cell and gene therapy. He is a respected leader in the field, serving in many roles on trade association and society committees. He recently completed a term as the Chair of the Regulatory Affairs Committee for the American Society of Gene and Cell Therapy. Dr Wonnacott has spoken and published extensively on topics related to the regulation of cell and gene therapies. Dr Wonnacott received his PhD from the Pennsylvania State University and his undergraduate degree from Brigham Young University.
Luis Pelloso
Executive Medical Director, Pharmacovigilance & Scientific Medical Affairs at
Luis Pelloso, MD, PhD, is the Executive Medical Director in Medical and Scientific Services within Pharmacovigilance at PPD. Currently, Dr Pelloso leads the Oncology Medical Monitoring globally. He has served as an oversight therapeutic MM lead for Hematology-oncology medical monitoring, as a Global Medical Director for more than 50 Phase I-III trials, provided strategic vision and business direction within the department for MM and PV services and cross-functional within Hematology-oncology and rare diseases for thirteen years. Dr Pelloso received his MD in 1997 from the Faculdade de Medicina do ABC, Santo André, Brazil. He did his residency in Internal Medicine in Federal University of Sao Paulo, Brazil (UNIFESP), where he also did his Fellowship in Hematology and received a PhD in Medicine in 2005.
Dan Takefman
Principal at Takefman Gene Therapy Advisors LLC
Daniel Takefman, PhD, is the Principal of Takefman Gene Therapy Advisors since March 2020. Dan provides expert regulatory advice for the development and commercialization of cell and gene therapies. Dr Takefman also assists in due diligence assessments of gene therapy assets for venture capital firms and is a member of multiple scientific advisory boards.
Previously Dr Takefman was SVP and Head of Regulatory Affairs at Spark Therapeutics for 5 years. At Spark, Dr Takefman supervised the submission through to approval of the FDA and EMA Luxturna® marketing applications. Dan also supervised the regulatory process for multiple AAV based investigation products including two additional Breakthrough Designation products: SPK-9001 for the treatment of Hemophilia B (marketing applications currently under review by FDA and EMA) and SPK-8011 for the treatment of Hemophilia A.
Dr Takefman joined the FDA Division of Cellular and Gene Therapy in 1999 as a Postdoctoral Fellow. Dan held multiple positions during his 15-year career at FDA, ultimately becoming Chief of the Gene Therapy Branch. He supervised the Chemistry, Manufacturing and Control (CMC) review process for all gene therapy products and for a variety of therapeutic vaccine products. Dr Takefman holds a PhD in Immunology from Rush University and a BS in Microbiology from the University of Iowa.
Elizabeth Donahue
Senior Director, Peri-and Post-Approval Studies & Real World Evidence at
Elizabeth Donahue is a Senior Director of Project Management in the Peri- and Post-Approval Studies and Real-World Evidence team. She brings over 17 years of CRO experience to her position. As an operational subject matter expert, Elizabeth is instrumental in the strategic delivery of trials with varying study design, including Phase III clinical trials, observational studies, registries, chart reviews, and long-term follow-up studies. Her therapeutic experience focuses on rare diseases, pediatric studies and cell and gene therapies, which encompasses multiple indications in endocrinology, hematology, neurology and more. Elizabeth is also a member of CRG’s Rare Disease and Pediatrics Center of Excellence, a group dedicated to delivering expertise and supporting clinical study teams across a variety of rare indications and those affecting children.
Orin Tempkin
Global Regulatory Leader/ Senior Director at Johnson & Johnson Innovative Medicine
Orin Tempkin is Global Regulatory Affairs Leader, Senior Director, in the Retina therapeutic area at Johnson & Johnson Innovative Medicine, based in New Jersey. He and his team oversee the development of botaretigene sparoparvovec, an investigational gene therapy currently in Phase III clinical trials for treatment of an inherited retinal disease. Prior to 2021, at Novartis, he worked on the international registrations of Luxturna™ (voretigene neparvovec). With more more than 20 years in regulatory affairs, Orin has previously developed and registered therapies in a number of disease areas including respiratory, infectious disease, and cardio-metabolic. His passion for meeting the needs of patients with rare diseases originated with his work on cystic fibrosis programs. Orin holds a PhD in organic chemistry from MIT.
