ALBUQUERQUE, N.M., Nov. 16, 2021 -- Gene Therapy has enjoyed exponential growth over the past decade due to the evolution of the technology, significant investment in the biotechnology sector, and the emergence of approved drugs that have broken through an important barrier. Due to both clinical and some nonclinical reports, the FDA has raised some concerns related to potential safety risks with new treatments, including immune and neurological consequences.
As drug developers at contract research laboratories, we benefit from monitoring FDA review and input into a wide range of study designs, indications and disease areas, which informs the organization and execution of our own studies. This presentation will highlight and summarize common threads that have been observed in terms of FDA requests for neurological endpoints as part of the nonclinical package. We will define aspects of the protocols that are employed and provide examples of data where appropriate. The presentation will include: dose considerations, neurobehavioral assessment, necropsy collections for biodistribution and histology, and histological outcomes.
Tuesday, December 07, 2021
12:00 PM Eastern Time
Gene Therapy Toxicology Fact Sheet
Dr. Jake McDonald is a Senior Scientist and Vice President of Applied Science at Lovelace Biomedical. In this role he serves as Director and Technical Advisor to collaborators in the design and execution of nonclinical development packages related to toxicology and pharmacology. Much of his emphasis is in the development of Gene Therapy technologies under GLP to support IND submissions. Dr. McDonald will present on the framework for the use of rare disease models and regulatory considerations for safety.
Dr. Jane Lindborg is a Study Director at Lovelace Biomedical with specialized training in gene therapy and neuroscience. She received her PhD in Neuroscience at Case Western and completed a Postdoctoral appointment at Yale in gene therapy applied to neurological models. At Lovelace Dr. Lindborg conducts neuropharmacology and toxicology studies with both rodents and nonhuman primates. She will speak to the design considerations and protocols for the assessment of neurological liabilities.
Dr. Debra Tokarz is a veterinary pathologist at Experimental Pathology Laboratories (EPL), Inc. She provides pathology support for commercial and academic toxicology studies and the National Toxicology Program. This support includes gross and microscopic pathology evaluation for GLP and non-GLP safety, efficacy, and investigative studies, as well as study design and protocol development. Dr. Tokarz has expertise in neuropathology and performs pathology evaluations for rodent and non-rodent gene therapy studies. She will present on sampling considerations and some common findings histologic outcomes.
About Lovelace Biomedical
Lovelace Biomedical is a contract research organization that conducts research to advance pharmaceutical and biotechnology companies in their complex drug development studies from the preclinical stage, and on to clinical trials. For over 70 years, the organization has leveraged its multidisciplinary expertise in toxicology, gene therapy, neurological disorders, infectious disease, and medical countermeasures.
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About EPL Inc.
Founded in 1971, Experimental Pathology Laboratories, Inc. (EPL®) is a private, employee-owned U.S. company recognized internationally for its ability to meet the needs of the scientific community and the challenges of preclinical safety testing with high-quality, on-time histopathology services.