WHAT IS THE NHLBI GENE THERAPY RESOURCE PROGRAM?

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.