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 Clinical Vector Core provides manufacturing of clinical and pre-clinical Adeno-Associated Virus (AAV) vectors of serotypes 1, 2, 5, 6, 8, 9, and Lentivirus (LV) vectors. Novel or modified serotypes will require development prior to scale-up. Products for clinical use are manufactured in compliance with Current Good Manufacturing Practices (cGMP) for Phase 1 and 2 clinical trials. To support pre-clinical work, including pharmacology and toxicology studies, we offer products manufactured using a GMP-comparable process. Research-grade products for proof-of- principle and bridging studies are also offered.