The Raymond G. Perelman Center for Cellular and Molecular Therapeuticsat The Children's Hospital of Philadelphia has established a state-of-the-art cGMP clinical vector manufacturing suit for both adenoassociated and lentiviral vectors. The facility is under the direction of Han van der Loo, a leading expert in clinical vector production and characterization. Our goal is to help to realize the enormous promise of gene transfer therapy to address unmet medical needs.
The Core Facility uses a patented vector production technology and a highly efficient purification process that uses combined column and gradient centrifugation-based process steps. This system has manufactured clinical-grade AAV vectors that have demonstrated excellent safety in several clinical studies. We are also in the process of implementing GMP Lenti vector production.
Our production efficiency is achieved along with exceptionally high vector product quality. Our final product is highly purified, containing negligible amounts of impurities such as empty capsid. Prior to the final release of the clinical vectors, the vector product is subjected to numerous tests to ensure quality. We also provide federal regulatory documentation and necessary certifications.
Recently, the Clinical Vector Core has also installed Lenti viral vector production capacity. Drawing from both in-house and newly acquired expertise, our Lenti group has quickly implemented an efficient production process that can meet our collaborators' clinical protocol needs.
Our proven track record, consistency, high vector production capacity and world-class expertise have enabled us to achieve national recognition. We were awarded a prestigious five-year NHLBI contract to assist in vector design, cGMP manufacture, and certification of AAV vectors.
Nasimuzzaman, M., Lynn, D., Ernst, R., Beuerlein, M., Smith, R.H., Cross, S., Link, K., Lutzko, C., Nordling, D., Russell, D.W. et al. (2015) Production and purification of high-titer foamy virus vector for the treatment of leukocyte adhesion deficiency. Mol Ther Methods Clin Dev, submitted.