Gene therapy investigators can greatly benefit from National Gene Vector Biorepository

Reviewed by Emily Henderson, B.Sc.Mar 27 2020

Gene therapy investigators can greatly benefit from the resources and services provided by the National Gene Vector Biorepository (NGVB), housed at the Indiana University School of Medicine. These include 93 unique reagents, a searchable database of animal safety studies, and Replication Competent Virus Testing, as detailed in an article published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.

"The National Gene Biorepository: Eleven Years of Providing Resources to the Gene Therapy Community." was coauthored by Kenneth Cornetta, Lorraine Matheson, Ryan Long, and Lisa Duffy, Indiana University School of Medicine, Indianapolis.

The Reagent Repository has distributed more than 1,000 reagents to nearly 400 investigators. Included in the repository are reagents such as plasmids for adeno-associated virus (AAV) gene transfer and cell lines used to manufacture retroviral, lentiviral, adenoviral, and AAV gene therapies. Also included in the NGVB's resources and services is the Pharmacology and Toxicology (Pharm/Tox studies) Good Laboratory Practice (GLP) archive for storing samples from pharm/tox studies.

The authors describe three main rationale for offering NGVB services: decreasing the risk of non-compliance with FDA requirements; addressing the disconnect between clinical trial grant funding periods and extended post-trial monitoring requirements; and reducing costs.

The NGVB resource is one of the key elements in the NIH strategy to enable academic researchers to pursue early phase clinical trials of their gene therapies with or without industry involvement. Without such resources, the cost of these services would otherwise be prohibitive for academic scientists, and their work would perish in the proverbial 'valley of death'"

Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Worcester, MA