In June 2005 the NHLBI convened an expert Working Group to better understand the impediments to clinical progress in gene therapy and to advise the NHLBI how to best direct its limited resources to facilitate translational research in the field of gene therapy. The NHLBI responded to these recommendations by establishing the NHLBI Gene Therapy Resource Program (GTRP) in March 2007. The GTRP consists of a Clinical Coordinating Center (CCC) and four Core Laboratories. The Adenovirus-Associated Vector (AAV) Production Core Laboratory is charged with supplying preclinical and clinical-grade products to investigators in a manner that is compliant with all relevant regulatory requirements. The laboratory works very closely with investigators who request services from the GTRP, the Core Laboratories, and the NHLBI to ensure that all aspects of the GTRP run smoothly and that the work performed is within with the scope of the GTRP and the Mission of the NHBLI. During the initial funding period the AAV laboratory has conducted approximately 3-4 campaigns per year for 10-20 patient clinical trials. Two GMP-grade AAV vectors have been produced, and scale-up may be required if the products are approved for larger trials. Fulfilling the various service requests requires extensive interactions with all parties involved and the process can take months to years, particularly those aspects related to human clinical trials. Therefore, it is critical that the AAV laboratory have demonstrated experience in the management of complex research programs and human clinical trials.
