AAV Services
Adeno-associated virus (AAV) has become one of the most widely used vectors for gene delivery in both basic research and clinical gene therapy. Its unique combination of safety, stability, broad tropism, and long-term expression makes AAV an ideal vehicle for delivering therapeutic genes to targeted tissues.
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 services designed to accelerate research and therapeutic development. Our offerings include: (1) AAV vector design, packaging, and characterization; (2) AAV library construction, production, and evolution; (3) AAV capsid engineering and development. Additionally, we provide DNA barcoding technologies for efficient evaluation of promoters, enhancers, and capsid variants. We also offer comprehensive AAV experimental services both in vivo (mice, rats, NHP) and in vitro (cell lines, primary cells, and organoids). With these services, AAVnerGene is committed to empowering researchers and biotech companies to drive innovation and achieve success in AAV gene therapy development.
AAVnerGene provides comprehensive AAV vector design and AAV genomic optimization services, helping clients enhance AAV specificity, potency, and safety. Our experts guide the selection of appropriate AAV capsids, plasmid backbones, promoters, enhancers, and regulatory elements and labeling strategies to achieve precise transgene expression in target cells and tissues.
Plasmid Design: AAVone, AAVdual and AAVtri
- AAVone® (single-plasmid) system, capable of achieving AAV9 productivity exceeding 5e15 vg/L in HEK 293 cells.
- AAVdual® (two-plasmid) system, designed to balance productivity with flexibility.
- AAVtri, the smaller mini-pHelper plasmid (8.4kb) offers higher packaging efficiency.
Besides, we also provide AAV-shRNA and AAV-sgRNA design and screening services.
AAVnerGene delivers consistent, high-quality, and cost-effective AAV packaging services. Our proprietary AAV production systems (AAVone, AAVdual, AAVtri) allowing users to select the system that best fits their project requirements. Our proprietary transfection reagent, PEIone, enables highly efficient AAV vector production. AAVnerGene offers a range of cell lines (HEK 293T, HEK 293, and HEK293one) for AAV production, both adherent and suspension, which allows researchers to choose the most suitable system for their production needs. AAVnerGene provides iodixanol-based density gradients, cesium chloride (CsCl) based density gradients, and AAVx resin methods for AAV purification. Our extensive experience with both common and engineered AAV capsids, combined with comprehensive AAV analytical technology, enables us to consistently deliver high-quality AAV vectors.
Short Turnaround
2 weeks for ≤ 5E13GC
Guaranteed Titer
≥1E13GC/mL (qPCR-SYBR)
High Full Particle
≥80% by Mass Photometry
Good Quality
≥95% Purity
Large Capacity
up to 1E17 GC
Multiple Systems
AAVone/AAVdual/AAVtri
Abundant Serotypes
>1000 AAV Capsids
Extensive Experience
>5,000 delivered AAV vectors
Experienced Support
>20 Years AAV Experts
We offer essential quality control testing, including vector genome titration, infectious titer assessment, AAV capsid purity and component analysis, as well as endotoxin and mycoplasma screening. In addition, our advanced services cover AAV genome identity and integrity verification, empty/full particle ratio measurement, and genomic impurity analysis. Our proprietary AAV-Q platform enables rapid, accurate, and highly sensitive potency testing (TCID50) as well as replication-competent AAV (rcAAV) assays with exceptional efficiency. Together, these comprehensive tests provide vital insights into vector quality and safety, helping customers choose the most suitable AAV vector for their applications.
AAVnerGene offers comprehensive AAV library solutions—from custom AAV library design to high efficient AAV library construction and high-quality AAV library production, to multi-round directed evolution services in vivo and in vitro. Our platform supports a diverse range of AAV library types( AAV Capsid Library, AAV Capsid Barcode Library, AAV Promoter Library, AAV Enhancer Library, AAV-shRNA library, and AAV sgRNA library) tailored for discovery, optimization, and functional screening. With end-to-end capabilities and deep expertise in AAV engineering, AAVnerGene empowers researchers to accelerate innovation and uncover next-generation AAV vectors.
AAVnerGene’s ATHENA Capsid Engineering Platform includes three different sub-platform: ATHENA-I, ATHENA-II, ATHENA-III. By combining the three sub-platforms with AI, the ATHENA platform can efficiently identify, evolve and create the best AAV capsids for specific applications, potentially improving the effectiveness of gene therapy and reducing costs. AAVnerGene offers capsid selection and evolution services for both in vitro (primary cells, organoids) and in vivo (mouse, rat, NHP) models. We offer pre-made, high-complexity random peptide insertion AAV capsid libraries across multiple AAV serotypes, as well as AAV capsid barcode library services, enabling researchers to efficiently evolve and identify optimized AAV capsids.
AAVnerGene offers specialized AAV genome optimization services, designed to fit genome size to AAV packaging capacity, enhance the expression and functionality of your gene of interest (GOI) and reduce the impurities within AAV vectors, paving the way for successful gene therapy results. Those services includes:
- Genome Size Optimization: Optimize the size of your GOI and regulatory elements to fit within the AAV packaging limit (~4.7 kb). Use strategies such as small intron, minimal promoter, and compact polyadenylation signals to reduce vector size. Employ split gene or dual vector systems for larger transgenes that exceed the AAV packaging capacity.
- Cis-Element Optimization: Test and compare different regulatory elements (ITR, promoter, enhancer, intron, PolyA) to achieve optimal transgene expression levels and specificity. Comparison of large number of promoters and enhancers with barcode-seq technology.
- Codon Optimization of GOI: Refine the coding sequence of your GOI to enhance expression in the target cell type.
- Genomic Optimization: Refine the AAV genomic sequence to reduce snap-back, partial or other incomplete genomes and improve AAV quality.
- Backbone optimization: Refine the backbone sequences and reduce the backbone related impurities (such as resistant markers and bacterial origins) in final AAV products.
- Proven Expertise: With over 20 years of experience in AAV vector design and production, our team provides unmatched expertise in maximizing AAV yields.
- We utilize a variety of transfection reagents, including PEImax, AAV-Max, FectoVIR, PEIone (AAVnerGene), and others.
- Additionally, we optimize culture conditions—including cell density, media formulations, enhancers/boosters, and supplemental additives—to achieve the best production performance.
- Our production platforms include multiple producer cell lines such as HEK293T, HEK293, HEK293one(AAVnerGene), and more.
- We support diverse cell culture systems ranging from traditional flasks to large-scale bioreactors like the XDR200.
- Innovative Systems: Our proprietary AAVone, AAVdual, and AAVtri systems offer cutting-edge solutions for AAV production, giving you more options for efficiency and scalability.
- End-to-End Support: From initial consultation to testing, optimization, and scale-up, we support you throughout the entire AAV production process.
At AAVnerGene, we developed AAV-Q Platform, which works for both AAV Potency (TCID50) and rcAAV assay, providing a complete and standardized system for AAV infectivity and rcAAV analysis.
- Three Cell Lines:
- HEK293R: AAVR overexpression based on HEK 293A cells;
- HEK293RR: Rep overexpression based on HEK 293R cells;
- HEK293Rd: Optimized for Rd-Ad5 production;
- One Ad vector:
- Rd-Ad5：Replication deficient in HEK 293A, but not in HEK293Rd cells;
- Two Assays:
- AAV TCID50 Assay: Measures AAV potency or infectious titer using HEK293RR cells with Rd-Ad5;
- rcAAV Assay: Detects replication-competent AAVs using HEK293R cells with Rd-Ad5;
AAVnerGene offers comprehensive Neutralizing Antibody (NAB) testing services to accurately assess anti-AAV immune responses, critical for evaluating vector efficacy and patient eligibility. Our NAB Testing Options:
ELISA-Based NAB Assay
A sensitive and high-throughput method to detect total anti-AAV antibodies in serum or plasma samples.Cell-Based NAB Assay
A functional assay that measures the ability of antibodies to neutralize AAV transduction in target cells, providing direct insight into neutralizing activity.
Our expert team ensures reliable, reproducible results to support your gene therapy development and clinical trial programs.
Many AAV-related publications are difficult or even impossible to reproduce, and one of the major underlying reasons is variation in AAV vector quality. AAV preparations can differ significantly from lab to lab and from batch to batch, even when the same serotype, transgene, and protocol are used. Without rigorous AAV vector validation, researchers may unknowingly compare results generated with vectors that differ in genome integrity, full-to-empty capsid ratio, impurity levels, or functional activity. These hidden differences often result in inconsistent in vitro and in vivo outcomes, misleading dose–response relationships, and poor reproducibility across studies. AAVnerGene offers comprehensive AAV validation and AAV biodistribution services to support all stages of your AAV research projects. Our expertise spans from in vitro studies to advanced in vivo models, ensuring robust evaluation of AAV vectors across relevant biological systems. Our Experimental Service Capabilities Include:
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AAV Vector validation: Our platform covers every critical aspect of vector characterization, including AAV capsid structure and AAV purity, AAV full-to-empty particle ratio, AAV physical titer and AAV infectious titer, AAV genome integrity.
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In Vitro Cell Models: Transduction and functional assays in diverse cell lines to assess AAV tropism, expression, and potency.
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Human Organoids: Cutting-edge 3D culture systems to study tissue-specific gene delivery and biological effects in physiologically relevant contexts.
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In Vivo Rodent Models: Comprehensive AAV evaluation in mice and rats, including biodistribution, transgene expression, and efficacy studies.
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Non-Human Primate (NHP) Studies: Advanced preclinical testing of AAV vectors in NHPs to assess translational potential, safety, and tissue targeting.
With AAVnerGene’s full-spectrum experimental services, you can accelerate your gene therapy development with confidence and precision.