Clinical Vector Core

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The Raymond G. Perelman Center for Cellular and Molecular Therapeutics (CCMT) Clinical Vector Core is a non-profit facility that provides state-of-the-art current Good Manufacturing Practices (cGMP) clinical vector manufacturing of adeno-associated virus (AAV) and lentiviral (LV) vectors. Johannes van der Loo, PhD, a leading expert in preclinical and clinical vector production and characterization, directs the Clinical Vector Core with the goal of helping to realize the enormous promise of gene transfer therapy to address unmet medical needs.

The Core provides platform manufacturing for LV vectors and for a variety of standard AAV serotypes, including 1, 2, 5, 6, 8, and 9. Novel or modified serotypes may require a pilot run prior to scale-up. Products for clinical use are manufactured in compliance with FDA cGMP regulations for Phase 1 and 2 clinical trials. To support pre-clinical pharmacology and toxicology studies, we offer GLP-grade products manufactured using a GMP-comparable process. Research-grade products for proof-of-principle and bridging studies are also offered. Finally, we provide testing services for long-term stability and device compatibility studies and provide Chemistry, Manufacturing and Control support for Investigational New Drug and Investigational Medicinal Product Dossier applications.

The Children’s Hospital of Philadelphia Clinical Vector Core is an approved supplier of investigational products for the National Institutes of Health (2021) and welcomes national and international projects from academia, industry, and government.