SIRION adeno-associated virus (AAV) vector services

SIRION Biotech’s AAV services include development of custom adeno-associated virus vectors that can be used for basic research or preclinical drug development.

AAVs are seen as the gold standard for in vivo expression and knockdown experiments, thanks to their unique biology, including lack of pathogenicity and abilities to infect non-dividing cells.

Addressing the entire AAV value chain

Successful gene therapy approaches necessitate exact gene delivery and expression control, suited to the highly complex in vivo environment of clinical applications. By serving the entire AAV value-chain, SIRION provides a rapid gateways to the best clinical vectors, with optimization across all three tiers of successful AAV implementation:

• Transcription: Customized vector design including promotor choices and enhancer elements to guarantee optimal expression of your gene in the targeted tissues.
• Transduction: AAV capsid mutations and surface variant constructions to optimize the tissue tropism. Includes options of directed evolution through peptide insertion, shuffled and barcoded AAV libraries.
• Immunogenicity: AAV capsid surface modulation to optimize/minimize the immunogenic profile of the selected AAV strategy.

SIRION Biotech offers full project management to invent new, individually optimized AAV vectors, with knowledge of all necessary patent framework to enable the creation of new intellectual properties.

End-to-end AAV service

SIRION offers expert AAV production and engineering services, providing high quality products within as little as one month. The sophisticated SIRION platform allows virtually any desired expression construct to be integrated within the vector’s cloning capacity.

The full SIRION AAV service includes:

• Cloning of cDNA/ shRNA into expression vector with free choice of promoter
• Optimization of codon usage for targeted expression
• DNA-sequencing verification of cloning success
• Small-scale test production with Go/ No-Go decision for full production
• Production: Transfection of 293T cells with customized expression vector and serotype specific helper plasmids
• One or two-step purification and concentration
• Genomic titer determination, QC and Delivery

Batch sizes can range from 1×10¹² VG (vector genomes) for small explorative experiments up to 5×10¹⁴ VG to satisfy complete experimental cohorts.

AAV Optimization for Gene Therapy and Clinical Trials

Additional in-house features of the SIRION Biotech AAV technology platform include surface modified particles for cell-specific transduction. Every project is conducted in close liaison with the customer to ensure that the resulting particles are feasibly designed to fit the final application perfectly.

The success of AAV in vivo experiments depends on vector quality,  titer and purity, along with correct choices of serotype and promoter.

A special feature of the SIRION AAV services is the manufacture of a range of AAV serotypes in combination with tissue specific promotors to further increase gene expression and precision.

Custom AAV service

SIRION Biotech’s custom AAV service includes technical assistance to identify optimal settings for specific application so that AAV vectors can be engineered to exact customer specification.

The customized service harnesses SIRION Biotech’s keys to success:

• Vector evolution and engineering
• Established manufacturing processes, from R&D into clinic
• Strong client commitment with designated multi-lingual contact managers
• Streamlined communication through strategic partnerships with academic & CDMO partners
• Seamless process transfers

Resources

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