The Children’s Hospital of Philadelphia was last week awarded continuation funding from the National Heart, Lung and Blood Institute (NHLBI) for work as the Adeno-Associated Virus (AAV) Vector Production Core Laboratory in support of the Gene Therapy Resource Program (GTRP).
The GTRP was initially established in 2007 to better understand obstacles to clinical progress in gene therapy and prioritize utilization of limited resources to facilitate translational research. The GTRP, directed by the NHLBI Gene Therapy Group, consists of three vector production cores, a pharmacology/toxicology testing core, and a clinical coordinating center. The primary objective of the Adeno Virus Core Laboratory is to manufacture and certify AAV vectors for clinical use.
The core lab produces large-scale, clinical grade vectors (both viral and non-viral) for use by NHLBI-funded investigators. Vectors may include retroviruses, lentiviruses, adenoviruses, adeno-associated viruses, and non-viral plasmid vectors.
The AAV Vector Core Lab is currently in the process of manufacturing the following vectors:
AAV8 (retinoschesin gene) for juvenile retinoschisis
AAV8 (human GAA gene) for Pompe Disease
AAV 6 (SERCA 2a gene) for heart failure
AAV9 (car-diomyocyte sensor protein gene) for heart failure
AAV2 (neurotropic factor gene) for Parkinson’s disease
The contract award amount is $5,147,636. The funding activity was conducted under Solicitation Number: NIH-NHLBI-HV-12-08.
