Table 1. Institutions and rAAV GMP manufacturing technologies.
| Center, location | Production | Cells and platform | Purification | Serotypes | Removal empties | Reference |
|---|---|---|---|---|---|---|
| Powell Gene Therapy Center, Human Applications Laboratory, University of Florida, Gainesville, FL | 2-plasmid Transfection (CaPO4) | HEK293 CellSTACKS | Cell harvest microfluidization or acidic flocculation and lysis, Benzonase, Heparin AC, IEC: SP, POROS, PS, HA, Hollow fiber tangential flow filtration (TFF) | 1, 2, 2(Y444, 500, 730F), 9 | No | 14,48,51,56,63–70 |
| Center for Cellular and Molecular Therapeutics, Children’s Hospital of Philadelphia, PA | 3 plasmid Transfection (CaPO4) | HEK293 Roller Bottles | Cell and media harvest, TFF and microfluidization, Benzonase, Poros 50HS, CsCl gradient, TFF | 2 | Yes | 13,60,71–77 |
| Avigen Incorporation, Alameda, CA | 3-plasmid Transfection (CaPO4) | HEK293 | Cell harvest, PEG precipitation, CsCl gradient, Dialysis and concentration | 2 | Yes | 61,78–80 |
| St Jude Children Hospital Children’s GMP, Memphis, TE | 2-plasmid Transfection (CaPO4) | 293-T CellSTACKS | Cell harvest microfluidization, Benzonase, Sephacryl, Poros 50HQ, Sephacryl, TFF | 8 | No; then Yes | 8,50,81,82 |
| Belfer Gene Therapy Core Facility, Weill Cornell Medical college, New York, NY | 2-plasmid Transfection (CaPO4) | 293-T CellSTACKS | Cell harvest freeze-thaws, Iodixanol, Heparin affinity, Dialysis | 2, rh10 | Yes | 83–86 |
| Neurologix, Ft Lee, NJ | Transfection | HEK293 | Heparin affinity chromatography | 2 | 87,88 | |
| Asklepios Bio-Pharmaceuticals and University of North Carolina, Vector Core, Chapel Hill, NC | 3-plasmid Transfection (PEImax) | Suspension HEK293 Shaker flasks and WAVE Bioreactors | Cell harvest, Sonication, Benzonase, Clarification, Iodixanol gradient, Anion exchange chromatography, Diafiltration | 2, 2.5, 2i8, 8, 9 | Yes | 15,89–91 |
| Harvard Gene Therapy Initiative, Boston, MA | Transfection (CaPO4) | HEK293 CF-10 flasks | Freeze/thaw, Benzonase, Detergent, Iodixanol, Chromatography | 1, 2 | 92 | |
| Ceregene Inc, San Diego, CA | Transfection (CaPO4) | HEK293 | Heparin Affinity | 2 | No | 93 |
| Targeted Genetics Corporation, Seattle, WA | wtAd5 Infection | HEK293 Producer HeLa Cell line WAVE and stir Tank bioreactors | Depth filtration, Benzonase, Ion exchange, UF/TFF, Chrom step, Heat inactivation, Chrom step, NanofiltrationPolishingFormulation | 1, 2 | 94 | |
| AGTC, FL at SAFC, CA | HSV Infection | sBHK WAVE bioreactors | Detergent lysis, Benzonase, Depth filtration, TFF, Ion Exchange chromatography, Affinity chromatography, TFF | 1 | No | 38,41,42,95,96 |
| UniQure/AMT | Baculovirus Infection | Sf9 WAVE bioreactors | Proprietary | 1 | 5 |
The information in the table was assembled utilizing information gathered in detailed references or from general websites.