NHLBI-Funded Programs Provide Low-Cost Services to Gene Therapy Researchers

Preclinical and large animal studies can be costly, labor intensive, and time consuming. Smaller academic laboratories do not necessarily have the expertise or resources in house to perform such studies; obtaining services through CROs can be prohibitively expensive. While we now have the expertise to do these studies at my institution, at the time that I was performing studies in sheep and nonhuman primates, we needed the expertise of external research sites. I was new to large animal studies at the time and could have navigated many difficulties with the help of programs funded by NIH’s National Heart, Lung, and Blood Institute (NHLBI).

During the pandemic, I’ve explored the educational content from the National Gene Vector Biorepository (NGVB), which I’ve found to be extremely useful in planning our next experiments. Located at the Indiana University School of Medicine and funded by the NHLBI, the NGVB provides various resources and services that are helpful to investigators planning or performing a range of gene therapy studies. In addition to educational content, available services include archiving of study materials and samples, and a repository from which investigators can request reagents. The NGVB’s most sought-after service has been testing for replication competent viruses. “To date, we have supported almost 100 clinical trials in meeting this FDA requirement,” notes Kenneth Cornetta, M.D., Director of the NGVB. Other resources include a Pharm/Tox database with summaries of gene therapy Pharm/Tox studies on file with the FDA and a useful list of additional resources, FDA guidance documents, and organizations providing valuable services for investigators coordinating clinical translation. New services recently added include vector copy number and insertion site analysis.

The NGVB is one of several NHLBI-supported programs that provide low- to no-cost resources enabling translation of gene therapy research into clinical products. Other beneficial NHLBI-supported programs include the Gene Therapy Resource Program (GTRP) and the Primate Center for Gene Therapy. The GTRP can provide preclinical vector production through a Preclinical Vector Core at the University of Pennsylvania, pharmacology/toxicology testing through a Pharmacology/Toxicology Core at Lovelace Biomedical and Environmental Research Institute and regulatory support through a Clinical Coordination Center at Social & Scientific Systems, Inc. Also provided on the GTRP website are lists of funding, regulatory, and training resources. The Primate Center for Gene Therapy, located at UC Davis, provides support and services for preclinical gene therapy research studies, particularly in nonhuman primates across all age groups. While these programs are supported through the NHLBI, and services are primarily reserved for investigators studying heart, lung, and blood diseases, some do not have that requirement. Check the websites of each program for eligibility requirements and more information.

Dr. Pfister is assistant professor at the University of Massachusetts Medical School and chair of the ASGCT Communications Committee.

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