AAV Payload Engineering & Vector Design

The core provides payload engineering support to optimize vector performance for specific experimental and biological contexts.

What we support:

Transgene & cassette design

• Transgene cassette design and optimization
• Codon optimization and transgene size assessment

Regulatory elements

• Promoter selection (ubiquitous, tissue-specific, inducible)
• Regulatory elements (WPRE, polyA variants, introns)

Genome architecture

• ssAAV vs. scAAV design guidance
• Packaging constraints and genome size limits

Advanced payloads

• Design considerations for genome editing payloads (e.g., CRISPR/Cas systems)

Design consultation

Design consultations are available to ensure compatibility with selected serotypes, packaging limits, and downstream applications.

AAV Manufacturing & Characterization

We offer high-quality, small-scale AAV manufacturing optimized for research, preclinical, and early translational applications—without the overhead of large CDMOs.

Core Capabilities:

AAV Production

• Supported serotypes: All standard serotypes (engineered serotypes upon request)
• Production scale: 500 mL – 5 L suspension culture
• Expression system: Triple-plasmid transient transfection
• Custom transgenes: GOI, promoters, and regulatory elements

Purification & Downstream Processing

• Chromatography-based purification (affinity and anion exchange workflows)
• Scalable, clinically relevant processes
• Buffer exchange, formulation, and user-specified aliquoting

Quality Control & Characterization

• Vector genome (vg) titer (ddPCR)
• Capsid protein analysis (SDS-PAGE / Western blot)
• Full vs. empty capsid assessment (mass photometry, ddPCR)
• Endotoxin testing

Why Choose Us

Designed for Small, Medium, and Large-Scale & Rapid Turnaround

• in vitro proof-of-concept studies
• in vivo pilot and POC experiments
• Serotype comparison and process development
• IND-enabling groundwork

Translationally Relevant Processes

• Processes designed to minimize changes during clinical manufacturing

Scientific Expertise

• Expertise in serotype-specific purification behavior
• Chromatography process development
• Gene therapy vector engineering
• Collaborative problem-solving for difficult serotypes or novel constructs
   

Typical Deliverables

• Purified AAV vector (aliquoted, stored at −80 °C)
• Certificate of Analysis (CoA)
• Detailed production & purification summary
• QC data package

Applications

• Gene therapy research
• Genome editing (CRISPR/Cas delivery)
• in vivo functional studies
• Preclinical efficacy and biodistribution studies

Engagement Model

• Rapid project initiation
• Typical turnaround: 2–4 weeks
• Academic and startup-friendly pricing
• Full confidentiality and IP protection