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eLearning Seminar
Gene Therapy: Nonclinical and Regulatory Strategy

Speaker Bios

Joy Cavagnaro, PhD, DABT, ATS, RAC, FRAPS

Dr. Joy A. Cavagnaro is the President of Access BIO where she consults internationally on science-based preclinical development strategies for novel drug, biologics and device combinations. She received her PhD in Biochemistry from the University of North Carolina at Chapel Hill. Her career spans academia, the CRO and biotechnology industries and government. During her tenure at CBER/FDA she was appointed to the SBRS and served as FDA’s safety topic lead and rapporteur for “ICH S6.” She was the first to advocate the “case-by-case” rational science-based approach to preclinical safety evaluation. In 2011 she received SOT’s Biotechnology Specialty Section First Career Achievement Award and in 2019 the Society’s Arnold J. Lehman Award recognizing individuals who have made significant contributions to risk assessment and/or the regulation of chemical agents, including pharmaceuticals. She is Founder and Past Chair of the leadership committee of BioSafe, an expert preclinical science committee within BIO. She is a Past Chair of the Clinical and Regulatory Affairs Committee and Translational Science & Product Development Committee of the ASGCT. She was a member of the NTP Scientific Advisory Committee on Alternative Toxicological Methods and is currently an advisor and member of the Grants Working Group of the California Institute of Regenerative Medicine. She serves on multiple SAB’s and frequently lectures on translation and risk assessment of novel therapies. She is a past chair of CRRI, an independent investigational review board and is currently a member of Advarra IRB. She has co-authored numerous white papers, articles and book chapters related to various aspects of preclinical safety assessment. The book she edited “Preclinical Safety Evaluation of Biopharmaceuticals A Science-Based Approach to Facilitating Clinical Trials” published by John Wiley & Sons, NJ, 2008, is commonly referred to as the “BioBible”.

Tim MacLachlan, PhD, DABT

Dr. Tim MacLachlan is currently an Executive Director in the department of Preclinical Safety (PCS) at the Novartis Institutes for Biomedical Research in Cambridge, Massachusetts. Tim is the therapeutic area head within PCS for the Cardiovascular/Metabolic and the Liver/Kidney/Benign Hematology Disease Areas of NIBR. He also oversees safety assessment for biotherapeutics and gene and cell therapies within the NIBR portfolio. Prior to this, Tim held roles of increasing responsibility in the area of pharmacology and toxicology at Genzyme and Curagen. Tim has founded and participated in several industry and academic consortia focused on the nonclinical development of biotherapeutics and cell and gene therapies including chairing the nonclinical advocacy arm of the Biotechnology Innovation Organization (“BIOsafe), founding the Gene and Cell Therapy Committee for the European Federation of Pharmaceutical Industries and Associations (EFPIA).  Tim is also the EFPIA representative on the first International Conference for Harmonisation (ICH) guidance focused on gene therapy, ICH S12, directing conduct of gene therapy biodistribution studies. Tim received his PhD from Thomas Jefferson University and did his postdoctoral research with the Howard Hughes Medical Institute and the University of Pennsylvania.

Sandhya Sanduja, PhD

Dr. Sandhya Sanduja is currently a Team Leader in the Pharmacology/Toxicology branch of FDA/CBER/OTAT. Her work includes review of nonclinical data to inform regulatory and policy decisions for CBER advanced therapy products. Prior to joining the FDA, she was a postdoctoral fellow at the Whitehead Institute of MIT. Dr. Sanduja received her BS in Microbiology from Gargi College, New Delhi, India (2003), MS in Biotechnology from Jawaharlal Nehru University (2005), and PhD in Biological Sciences (2011) from University of South Carolina, Columbia. Dr. Sanduja has authored/co-authored several high-impact, peer-reviewed publications in leading biomedical journals, and received research grants from American Association for University Women (AAUW). She served on reviewer boards for PLOS ONE, Tumor Biology, and WIREs RNA and on the editorial board of Gastrointestinal Cancer. She is a member of the American Society of Gene and Cell Therapy.

Peter Ulrich, PhD, DABT, ERT

Dr. Peter Ulrich completed his study of Biophysics, Molecular Genetics, and Computer Science at the Universities of Freiburg at Albert-Ludwigs-Universität and Düsseldorf at Heinrich-Heine-Universität in Germany. His PhD thesis was on photoallergy as special case of contact dermatitis—investigation of the mechanism of the induction phase at the University of Düsseldorf. Dr. Ulrich completed his postdoctoral training in immunotoxicology at Bayer Research Center in Wuppertal, Germany. He has over 25 years in industry experience at Novartis as a lab/group head for immunotoxicology, hematology, phototoxicity as well as nonclinical safety assessment expert for low molecular weight drugs, antibodies, therapeutic vaccines, and gene therapy products. Dr. Ulrich holds board certifications from ABT and ERT. He is also a member of the Society of Toxicology, German Society of Toxicology, European Society of Gene and Cell Therapy, and German Society of Immunology.

Laurence Whiteley, DACVP, DVM, PhD

Dr. Laurence Whiteley earned his DVM from Purdue University, PhD from University of Minnesota, and did postdoctoral training at the Chemical Industry Institute of Toxicology. He is a Diplomate of the American College of Veterinary Pathology. Dr. Whiteley has more than 50 peer reviewed publications, book chapters, and patents in the area of veterinary pathology, product safety assessment and the discovery of novel therapeutics. He has 30 years of industrial experiences leading investigative programs evaluating mechanisms of toxicity and as the nonclinical drug safety lead on therapeutic candidate development teams. Dr. Whiteley has been involved in the use and development of viral vectors for target modulation in nonclinical studies and as therapeutic candidates for the past 14 years. He has made several invited presentations at national meetings on the nonclinical development strategy for gene therapies. Dr. Whiteley is PhRMA’s lead representative on the ICH S12 expert committee that is developing guidelines for the nonclinical biodistribution assessment of gene therapies. He is currently the nonclinical safety lead on two of Pfizer’s phase III clinical stage gene therapy programs, the Pathology Therapeutic Lead for Rare Disease, and serves as the Pathology and Investigative Toxicology Scientific and Strategic Advisor in the department of Drug Safety Research and Development at Pfizer.