The quality control requirements for AAV gene therapy drugs are stringent and challenging. Firstly, AAV vector drugs are more complex in structure and function than traditional small molecule drugs and protein biologics, and are more difficult to identify in terms of integrity and purity. Secondly, the production of AAV drugs is accompanied by very complex biological reactions, and a variety of impurities (e.g., cellular debris, heterologous nucleic acids, etc.) can be mixed in the products. The identification of various impurities requires the use of complex assays. Therefore, the development and application of a rigorous scientific quality methodology is an indispensable part of the process development and release of customized AAV gene drugs.
Genevoyager has developed systematic quality testing methods for AAV products, including product identification, purity testing, content titer and potency, impurities, safety, and physical inspection, thus ensuring consistent high quality of AAV at every stage under GMP conditions.
|
Category |
Quality Attributes |
Analytical Method |
|
Identification |
Capsid protein identification and purity |
SDS-PAGE |
|
Characteristic sequence identification |
First generation sequencing (target gene) |
|
|
Purity |
Capsid purity |
SDS-PAGE |
|
Aggregation |
SEC-HPLC |
|
|
Empty shell rate |
TEM, AUC |
|
|
Content and potency |
Physical titer |
ELISA |
|
Genomic titers |
qPCR,ddPCR |
|
|
Infection titer |
TCID50 |
|
|
specific activity |
Infectious titers, genomic titers |
|
|
Potency - Gene Expression |
Cell-based in vitro mRNA expression or protein expression（qPCR，ELISA，Western Blot） |
|
|
Potency-activity |
Cell-based in vitro biological activity, animal experiments |
|
|
Impurity |
Host cell DNA residues |
qPCR |
|
Nuclease residues |
ELISA |
|
|
Host cell protein residues |
ELISA |
|
|
Specific process-related residues (e.g., affinity ligands, PEI, decontaminants) |
ELISA,HPLC |
|
|
Security |
Bacterial endotoxins |
Gel limit test method |
|
Replicating viruses（rcAAV,BEV） |
Cultivation method |
|
|
Aseptic |
Membrane filtration method |
|
|
Mycoplasma |
Cultivation method，qPCR |
|
|
Viral exogenous factors |
Cultivation method |
|
|
Physical examination |
Appearance |
Visual method |
|
Packing quantity |
/ |
|
|
Visible Foreign Objects |
Light Inspection Method |
|
|
pH |
pH value measurement method |
|
|
Osmotic pressure |
Freezing point detection method |
|
|
Insoluble particles |
Photoresist method |
US: 3675 Market Street, Suite 200, Philadelphia, PA19104 Tel: +1 (215) 205-6963 | +086 027-65023363
E-mail: hui.wang@genevoyager.com
China: No128, Guanggu 7th Rd, East Lake High-tech Development Zone, Wuhan, China Tel: 17720522078
E-mail: marketing@genevoyager.com
US: 3675 Market Street, Suite 200, Philadelphia, PA19104 Tel: +1 (215) 205-6963 | +086 027-65023363
E-mail: hui.wang@genevoyager.com
China: No128, Guanggu 7th Rd, East Lake High-tech Development Zone, Wuhan, China Tel: 17720522078
E-mail: marketing@genevoyager.com
