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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:

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NHLBI Gene Therapy Resource ProgramMay 28, 2008Sonia I. Skarlatos, PhD, FAHAActing DirectorDivision of Cardiovascular DiseasesNHLBI Gene Therapy Coordinator

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Gene Therapy Resource Program Goals

  • Provide resources for NHLBI-funded investigators:

  • Provide regulatory assistance to NHLBI-supported investigators

  • Support gene therapy clinical trials

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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

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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

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Pharmacology/ToxicologyLovelace Biomedical and Environmental Research InstitutePI:Janet Benson, Ph.D.

Preclinical VectorsUniversity of PennsylvaniaPI:James Wilson, M.D., Ph.D.

Adeno-Associated Virus VectorsChildren’s Hospital of Philadelphia

PI:Fraser Wright, Ph.D.

Lentivirus VectorsIndiana UniversityPI:Kenneth Cornetta, M.D.

Gene Therapy Resource Program InfrastructureClinical Coordinating CenterSocial & Scientific SystemsPI:Micheal Gilbreath, Ph.D.

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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

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GTRP Contact Information

Sonia I. Skarlatos, Ph.D.

E-mail: SkarlatS@mail.nih.gov

Clinical Coordinating Center

E-mail: GTRPCCC@s-3.com

Web Site: www.gtrp.org