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 (https://www.nhlbi.nih.gov/about/strategic-vision). 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 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 a vector production core, 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.

Preclinical Vector Core

University of Pennsylvania

Produces large-, routine, and small-scale viral and non-viral based vectors for studies in basic research directed toward clinical applications and provides immunology services

Sample Immunology Testing RSA Sample Preclinical Vector Production RSA

GTRP page

Pharmacology/ Toxicology Core

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

Sample Pharmacology/Toxicology Testing RSA

GTRP page

Clinical Coordinating Center

Social & Scientific Systems, Inc. a DLH Holdings Corp [DLH] company

Coordinates RSA process management, Steering Committee management, regulatory support, and clinical trials funding assistance

Sample Clinical Trial Funding Assistance RSA Sample Regulatory Support RSA

GTRP page