Final Program - American Society of Gene & Cell Therapy

Program Schedule, Thursday, May 16, 2013
Scientific Symposium 215
10:30 am - 12:30 pm
ROOM: BALLROOM A
The GTRP: Perspectives of an Investigator and the Program Cores
CO-CHAIRS: Cheryl L. McDonald, MD and Sonia I. Skarlatos, PhD
SPEAKERS
Thursday, May 16, 2013
Daniel Rader, MD
How the GTRP Facilitates Translational Research: An Investigator’s Perspective
Atherosclerosis is a complex and heritable disease intimately inluenced by lipoprotein metabolism. Unbiased genome-wide
association studies (GWAS) have identiied dozens of novel loci robustly associated with both plasma lipid traits and atherosclerotic
coronary artery disease (CAD). Several of the genes at these loci could represent potential novel therapeutic targets for the
treatment of inherited dyslipidemias and the prevention or treatment of CAD. Adeno-associated viral (AAV) vectors are a valuable
tool for effective and stable in vivo expression of cDNAs in relevant animal models of disease. Through support from the GTRP, we
have used AAV8 vectors to express a number of potential therapeutic genes at lipid/CAD GWAS loci in the livers of ‘humanized’
mice. This systematic approach has not only yielded important new biological information about the effects of novel GWAS genes on
lipid metabolism and atherosclerosis, but also has placed a number of new ‘therapeutic’ genes on the map. This classic translational
approach moving from unbiased human genetics to mechanisms and therapeutic effects in model systems is made possible by the
support of the GTRP.
Sonia I. Skarlatos, PhD
GTRP: Facilitating Bench to Bedside
The purpose of the GTRP is to provide researchers with resources that are critical to advancing investigational gene therapy
products into clinical testing. Services along the product development spectrum – ranging from production of preclinical vectors to
assistance with early-phase clinical trials – are offered through the Program at no cost to the investigator. The Program primarily
supports research in heart, lung, and blood diseases.
James M. Wilson, MD, PhD
A Toolkit of Vectors for Gene Therapy
The Preclinical Vector Core provides high quality viral vectors based on adeno-associated virus (AAV), adenovirus and lentivirus
as well as non-viral vectors for basic research directed toward clinical application. Also offered are a host of other services such as
consultation on vector construction and experimental design, quality control services appropriate for vectors destined for use in
small or large animal models, immunology testing and preclinical biodistribution analysis. Of particular importance to NHLBI GTRP
investigators has been distribution through the program of novel serotype AAV vectors which have become the gold standards in
the ield for mediating gene delivery to heart, lung and liver. In addition to offering a variety of vector platforms, the Preclinical
Core rapidly transitions new technologies from key laboratories for distribution as well as develops reagents and maintains a large
inventory of vectors in order to provide GTRP participants with a veritable vector toolbox for advancing preclinical studies toward
clinical translation.
Janet Benson, PhD, DABT
From Mice to Man: IND-enabling Preclinical Studies
The mission of the Pharmacology/Toxicology (Pharm/Tox) Core is to aid investigators in the design and implementation of INDenabling
preclinical safety and biodistribution studies. This is often one of the most challenging and costly steps in the translational
process. The Pharm/Tox Core scientists work closely with the Investigator(s), the GTRP Core providing the vector (if applicable),
and the GTRP Clinical Coordinating Center to facilitate development of the IND packages and the pre-clinical study protocol. The
GTRP Pharm/Tox Core provides dosing, endpoint evaluations including but not limited to: clinical observations, clinical pathology,
histopathology, safety pharmacology, vector biodistribution (qPCR), immunology endpoints, as well as assessments of transgene
expression. Upon completion of the study, the Pharm/Tox Core staff prepares an audited inal report for inclusion in the IND
submission to the FDA.
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Final Program SALT LAKE CITY, UTAH May 15–18, 2013