Considerations to optimize and act upon long-term follow-up studies for cell and gene therapies

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.

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 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 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.