Non-clinical development of gene therapy: use of rare disease models, cystic fibrosis as a characteristic example
Posted: October 21, 2021 at 1:19 PM CDT|Update: 1 hour ago
ALBUQUERQUE, New Mexico, 21 October 2021 / PRNewswire / – Lovelace Biomedical, a contract research organization with a vast and rich history and expertise of over 70 years in the study of respiratory disease and the development of preclinical therapies, will present a webinar on gene therapies for disease, and using cystic fibrosis findings as a case study.
Discover the interactive multi-channel press release here: https://www.multivu.com/players/English/8964851-lovelace-biomedical-webinar-rare-diseases-gene-therapy-preclinical-research/
Non-clinical development to advance promising gene therapies often requires the use of new animal models to prove efficacy and / or safety. This webinar will define the basis for typical non-clinical study needs to advance gene therapies from concept to clinic; taking into account regulatory guidelines 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 will use CF as a typical example of the use of various animal model approaches to assess the safety and efficacy of therapies, including a discussion of the disease. administration of drugs and not pulmonary. safety assessment.
Register for the webinar here: https://www.webcaster4.com/Webcast/Page/2117/43201
Dr. Jake mcdonald is Senior Scientist and Vice President of Applied Sciences at Lovelace Biomedical. In this role, he acts as director and technical advisor to collaborators in the design and execution of non-clinical development packages related to toxicology and pharmacology. Much of its focus is on developing gene therapy technologies under GLP to support IND submissions. Dr McDonald will present the framework for using rare disease models and regulatory considerations for safety.
Dr. John engelhardt is professor and director of the Department of Anatomy and Cell Biology and director of the University’s Gene Therapy Center 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 diabetes and pulmonary diseases associated with cystic fibrosis, 2) the study of stem cells in the respiratory tract and the pancreas, 3) the development of animal models of cystic fibrosis. pulmonary and pancreatic diseases; and 5) the development of gene therapies for cystic fibrosis with an emphasis on recombinant parvoviruses and their transduction biology.
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 preclinical to clinical trials. For more than 70 years, the organization has used its multidisciplinary expertise in toxicology, gene therapy, neurological disorders, infectious diseases and medical countermeasures.
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