The Raymond G. Perelman Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia has established a state-of-the-art cGMP clinical vector manufacturing suite for both adeno-associated viral vectors and Lenti viral vectors, help to realize the enormous promise of gene transfer therapy to address unmet medical needs.
The Core Facility utilizes a patented vector production technology and a highly efficient purification process that utilizes 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.
Replication Competent AAV
Replication Competent LV
-- Manufacturing of clinical and non-clinical AAV and LV vectors per client specification
-- Long-term stability (3, 4, 5 years) consistent with the duration of the Phase 1/2 study
-- Device compatibility and short-term stability studies (as required)
-- Manufacturing of clinical or non-clinical excipient
-- Regulatory support for Investigational New Drug (IND) applications
-- Letter of cross reference to our Drug Master File (DMF)