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The NHLBI Gene Therapy Resource Program (GTRP) facilitates the translation of gene therapy research into clinical interventions. The GTRP provides resources for gene therapy research primarily in heart, lung, and blood diseases as reflected in the NHLBI Mission (http://www.nhlbi.nih.gov/about/org/mission.htm). Requests for resources for gene therapy research that are consistent with the missions of other NIH Institutes may also be considered by the Program.
Resources are provided in the form of preclinical and clinical-grade vector production, pharmacology/toxicology testing, immunology testing, clinical trials funding assistance, and regulatory support at no cost to the investigator. Investigators must first receive approval of their Registration with the Program in order to request resources.
The GTRP, directed by the NHLBI Gene Therapy Group, consists of three vector production cores, a pharmacology/toxicology testing core, and a clinical coordinating center. A Scientific Review Board and Steering Committee review Request for Service Applications and make recommendations to the NHLBI Gene Therapy Group regarding the applications’ scientific merit, feasibility, and compatibility with the Program’s mission.
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University of
Pennsylvania
Produces large-
and
small-scale
viral and
non-viral vectors for studies in basic research directed toward clinical applications; performs immunology testing
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The Children’s Hospital
of Philadelphia
Produces scalable
clinical-grade adeno-
associated virus (AAV)
vectors for use in clinical studies and GMP process-comparable
AAV vectors for use in pharm/tox studies |
Indiana
University
Produces scalable
clinical-grade lentiviral
vectors for use in clinical studies and GMP process-comparable lentivirus vectors for use in pharm/tox studies
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Lovelace Biomedical
and Environmental
Research Institute
Performs toxicology testing and
bio-distribution studies of
vectors in large and small
animal models as a prerequisite for use in clinical studies |
Social & Scientific
Systems, Inc.
Coordinates
RSA process management, Steering
Committee management,
regulatory support,
and clinical trials
funding
assistance
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