Toxicology Database

The National Gene Vector Biorepository offers an informational toxicology database as a resource to gene therapy investigators. The database contains information on animal studies that have been conducted to study the biodistribution of vector to different target organs and to evalutate potential toxic effects associated with the use of various vector systems. Studies within the database have been submitted to the US FDA in support of gene therapy clinical trials. The purpose of the database is to inform gene therapy investigators about the conclusions of prior toxicology studies and to facilitate the cross-referencing of relevant studies in support of new Investigational New Drug Applications (INDs) applications.

The studies are generally small, randomized single dose or dose escalation studies consisting of various animal models from mouse to non-human primate. The study parameters capture the test system, vector information, dose procedures, clinical observations, macroscopic/microscopic pathology, histopathology, tissue collection for PCR analysis, clinical pathology: hematology/chemistry analysis and a summary of the relevant findings.

These studies are paramount in moving from preclinical animal studies to clinical trial. With this in mind it is anticipated that by sharing this data, investigators in the gene therapy field may find a study in the database that is relevant to their specific vector and study of interest. Investigators can then utilize this data by acquiring a letter of cross reference from the Study PI allowing the FDA to review the data and assess whether a specific study would need to be conducted or whether there was no need to conduct the study.

Investigators have agreed to allow submission of their data into this database. The database is also accessible to any investigator in the gene therapy field who would like to share their data.

The database is being expanded to include non-GLP studies submitted to the FDA in support of an IND. These studies are conducted under careful compliance parameters and can be critical to platform studies in the development of novel vectors.

Pharmtox database is here.