AAV Experimental Services
AAVnerGene is pushing the boundaries of gene therapy with cutting-edge AAV technologies and solutions. Leveraging our team’s over 20 years extensive expertise, we provide comprehensive AAV experimental services, from inivtial vector design to final AAV gene drug development, designed to accelerate research and therapeutic development for AAV vectors.
| Service | Detailed Description / Deliverables |
| AAV Vector Validation & QC | Vector genome (VG) titer by qPCR/ddPCR, capsid titer, full/empty ratio, transgene integrity, promoter verification, and batch consistency |
| AAV Transgene Expression | In vitro (cell lines, primary cells and organoids) validation of transgene or reporter expression by fluorescence, luciferase, qPCR, or western blot |
| AAV Biodistribution Analysis | Quantitative tissue distribution analysis of AAV genomes and transgene expression across organs using qPCR/ddPCR and NGS |
| AAV Dose & Injection Route Optimization | Evaluation of dose response and administration routes (IV, IM, IVT, ICV, etc.) in special disease models (mouse, rat, NHPs) |
| AAV Genome Optimization |
Optimize the size of your GOI and regulatory elements to fit within the AAV packaging limit and achieve optimal transgene expression levels and specificity; refine the AAV genomic sequence to increase gene expression, reduce partial or incomplete genomes and improve AAV quality; Refine the backbone sequences to reduce the backbone related impurities. |
| AAV NAB Testing | High-throughput Elisa based antibody binding assays for seroprevalence screening and sample prioritization; Functional neutralization assays to determine true inhibitory NAB titers |
| AAV Productivity Testing | Systematic comparison of AAV production systems, plasmid designs, cell lines, transfection reagents, plasmid ratios, media, and culture conditions; Scale-Down & Scale-Up Studies by evaluating across adherent and suspension platforms from plates to bioreactors |
| AAV Potency Assay | Cell-based functional assays to measure AAV activity of with our AAV-Q platform |
| rcAAV Assay | Detection and quantification of replication-competent AAV for safety assessment use AAV-Q platform |
| shRNA Design & Screening | Rational shRNA design, synthesis, and screening using U6/H1 promoters or miR-30 scaffolds driven by tissue-specific Pol II promoters |
| sgRNA design & Screening | Rational sgRNA design, synthesis, and screening for different CRISPR-CAS systems |
| AAV Packaging Efficiency Testing | Evaluation of genome encapsidation efficiency and full/empty particle ratios |
| AAV Capsid Screening & Evolution | In vitro and in vivo capsid selection using barcoded libraries and NGS-based readouts |
| Tropism & Cell-Type Specificity Testing | Evaluation of capsid performance in specific tissues or cell types |
| AAV Impurity Analysis | Detection of empty capsids, host cell proteins, residual DNA/RNA, and process-related impurities |
| AAV Experimental Consultation | End-to-end experimental design support including vector design, capsid selection, dosing strategy, and data interpretation |
| Preclinical Strategy Support | Guidance for IND-enabling studies, assay selection, and translational planning |