What is the primary focus of the NHLBI Gene Therapy Resource Program (GTRP)?

The NHLBI Gene Therapy Resource Program was established to facilitate the translation of gene therapy research into clinical interventions primarily for heart, lung, and blood diseases consistent with the NHLBI Mission: (https://www.nhlbi.nih.gov/about/strategic-vision). Other disease areas may be considered.

How can I access GTRP services?

First, you must complete the online Investigator Registration Application Form on this NHLBI Gene Therapy Resource Program (GTRP) main web page under the Investigator Registration tab. Once you receive registration approval by the NHLBI Gene Therapy Group, you will have access to the Request for Service Application (RSA) forms via this GTRP main web page. For a registration to be considered for approval, an investigator must be conducting gene therapy research at an institution located in the United States.

Can I apply for GTRP services if I am conducting gene therapy research in disease areas other than heart, lung, or blood?

Yes, you can apply for GTRP resources once you have registered with the program and received approval to submit a Request for Service Application (RSA). If your research is outside of the mission of NHLBI but funded by another NIH Institute, the NIH Institute funding your research must be willing to transfer funds to NHLBI to cover the cost of the service. The NHLBI Gene Therapy Group would review your RSA and discuss this option with the Program Officer at the respective Institute.

Can I apply for GTRP services if my gene therapy research is not funded by NIH?

Yes. Your RSA will be considered if your gene therapy research is consistent with the NHLBI Mission (https://www.nhlbi.nih.gov/about/strategic-vision).

As an approved investigator in the program, do I have to pay for GTRP services?

No. If your Request for Service Application (RSA) is approved, the program resources are provided at no cost to the investigator.

As an investigator from an institution outside of the United States, can I apply for GTRP resources?

No. The investigator’s primary gene therapy research site must be located in the United States.

Is there a deadline for submitting a Request for Service Application?

No. RSAs are accepted on a continuous basis.

What is the timeframe for review of a Request for Service Application?

Review of RSAs for preclinical vector production, immunology testing, and regulatory support will be completed within 2 weeks.

Review and assessment of a final version of the RSA submission for pharmacology/toxicology testing and clinical trial funding assistance will usually be completed within 8 weeks.

See “RSA Review Process” in the Highlights section of this GTRP main web page.

As an approved investigator, can I submit a Request for Service Application for pharmacology/toxicology testing or clinical trial funding if my gene vector product was manufactured outside of the GTRP?

Yes. The various types of RSAs are independent of one another. An investigator may submit any type of RSA for a qualified gene therapy research project regardless of past or future utilization of GTRP resources for the project.

Does the GTRP offer immunology testing?

Yes. Immunology testing of preclinical samples is provided by the University of Pennsylvania (U Penn) Preclinical Vector Core Laboratory. For more information on the type of immunology tests available, visit the U Penn web page via this GTRP main page.