NHLBI Gene Therapy Resource Program May 28, 2008 Sonia I. Skarlatos, PhD, FAHA Acting Director Division of Cardiovascular Diseases NHLBI Gene Therapy Coordinator.

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NHLBI Gene Therapy Resource Program May 28, 2008 Sonia I. Skarlatos, PhD, FAHA Acting Director Division of Cardiovascular Diseases NHLBI Gene Therapy Coordinator

Gene Therapy Resource Program Goals Provide resources for NHLBI-funded investigators: Provide regulatory assistance to NHLBI- supported investigators Support gene therapy clinical trials

Core Laboratories Preclinical Vector Core: Produces large- and small-scale viral and non-viral vectors for studies in basic research directed toward clinical applications Clinical-grade AAV and Lentivirus Cores: Produce scalable clinical-grade AAV and lentiviral vectors for use in heart, lung, and blood clinical studies Pharmacology/Toxicology Testing Core: Performs toxicology testing and biodistribution studies for vectors in large and small animal models as a prerequisite for use in clinical studies

Clinical Coordinating Center (CCC) Manage and coordinate all GTRP activities in conjunction with NHLBI –Resource Application Process –Scientific Review Board –Steering Committee Develop and maintain GTRP database and website Provide regulatory assistance to NHLBI-supported investigators Administer clinical trial funds

Gene Therapy Resource Program Infrastructure Clinical Coordinating Center Social & Scientific Systems PI: Micheal Gilbreath, Ph.D. Pharmacology/Toxicology Lovelace Biomedical and Environmental Research Institute PI: Janet Benson, Ph.D. Preclinical Vectors University of Pennsylvania PI: James Wilson, M.D., Ph.D. Adeno-Associated Virus Vectors Children’s Hospital of Philadelphia PI: Fraser Wright, Ph.D. Lentivirus Vectors Indiana University PI: Kenneth Cornetta, M.D.

Application Receipt Dates (starting in 2008) Preclinical Vectors/Regulatory Assistance: Any time during the year Pharmacology/Toxicology studies: February 15 and September 15 Clinical-grade vectors: February 15 and September 15 Clinical Trials Support: February 15 and September 15

GTRP Contact Information Sonia I. Skarlatos, Ph.D. Clinical Coordinating Center Web Site: