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Lovelace Biomedical Presents A New Webinar On Rare Diseases And Gene Therapy Preclinical Research

Gene Therapy Nonclinical Development: Use of Rare Disease Models, Cystic Fibrosis as a Hallmark Example

ALBUQUERQUE, N.M., Oct. 21, 2021 /PRNewswire/ -- Lovelace Biomedical, a contract research organization, who has a vast and storied, 70 plus year history and expertise in studying respiratory diseases and developing therapeutics at the preclinical stage, will present a webinar in gene therapies for diseases, and will use Cystic Fibrosis results as a case study.

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Nonclinical development to advance promising Gene Therapies often require the use of novel animal models to prove efficacy and/or safety. This webinar will frame the basis of typical nonclinical study needs to advance gene therapies from concept to clinic; with consideration of regulatory guidance and science in the design of experiments that may be considered by the FDA. The presentation will frame the overall regulatory and scientific constructs that are 'typical', and use cystic fibrosis as a hallmark example of the use of various animal model approaches to evaluate safety and efficacy for therapeutics, including a discussion of drug delivery and non-pulmonary safety evaluation.

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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. John Engelhardt is Professor and Chair of the Department of Anatomy and Cell Biology and Director of the Center for Gene Therapy at the University of Iowa Roy J. and Lucille A. Carver College of Medicine. His research focuses on the following areas: 1) the study of the molecular and cellular pathogenesis of cystic fibrosis (CF) related diabetes and lung disease, 2) the study of airway and pancreatic stem cells, 3) the development of animal models of CF lung and pancreatic diseases, and 5) the development of gene therapies for CF with a focus on recombinant parvoviruses and their transduction biology.

About Lovelace BiomedicalLovelace 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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