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Correcting Defective Genes to Cure Genetic Diseases

Successful gene therapy approaches necessitate exact gene delivery and expression control, suited to the highly complex in vivo environment of clinical applications. They also require effective cost control at all development and production stages. By serving the entire AAV value-chain SIRION Biotech is the gateway to fast, cost-effective clinical vectors

The key to SIRION’s integrated support is the Viral Vector Know-How Hub. The Viral Vector Know-How Hub, built up over more than a decade, is composed of proprietary technological assets and connects the expertise and resources of internal and external stakeholders. The mission of the hub is to develop optimized viral vectors and technologies with increased efficacy and patient safety.

The level of support the SIRION can provide helps de-risk cell and gene therapy development programs by:

Reducing development time

  • Addressing key clinical requirements– safety, efficacy/potency, manufacturability – as early as possible
  • GMP compliant process development and optimization for seamless process transfer into clinics via a network of dedicated CDMO partners

Robust platform processes

  • Manufacturing high quality and high titer preclinical batches up to 50L

Inside the AAV Viral Vector Know-How Hub

Technical and Scientific Capabilities of the Hub Development Engine

AAV Capsid Engineering

AAV directed evolution to isolate novel capsids

  • Library generation
  • NGS characterisation of libraries
  • Screening, biodistribution and PoC studies in large animals, including NHP via CRO network

Nanobody redirected targeting

  • Insertion of target cell surface protein binding nanobodies in AAV VP protein
  • Enhanced specificity and transduction levels

AAV Lead Vector Development

  • In-depth project analysis by SIRION gene therapy specialists
  • AAV vector expression cassette development for high on target and minimal off-target expression
  • Clinically compliant plasmid backbone for AAV production

AAV Vector Manufacturing

  • Process development for optimal productivity and negligible process or product-related impurity levels
  • Upscaling to 50 L
  • Customizable process development for accelerated transfer to CDMO Network for GMP manufacturing
  • Extended QC package compliant to regulatory guidelines

GMP-ready Drug Substance Blueprint

  • Vector and particle documentation
  • PoC and biodistribuition studies of the lead candidate batches with a CRO network
  • Transfer of Drug Substance and processes to GMP network

 Hub CDMO Network

Support for GMP Manufacturing

During the early R&D and preclinical project stages at SIRION, relevant individual project aspects are considered from the start to ensure a seamless clinical project transfer. SIRION has developed a global CDMO network over the last 5 years, and can recommend and introduce CDMOs worldwide that best suit customer location and individual project requirements.

Moving projects from the preclinical phase to a GMP maunfacturing facility can require set up of entirely new processes and analytics. SIRION services accelerate the preclinical phase while developing appropriate upstream and downstream processes and analytic tools to enable a smooth Project Transfer – speeding up the project transfer and process development phase at a CDMO by up to 8 months.


New Literature! Supporting Gene Therapy Development with Safer, More Efficiacious AAV Vectors 

Contact us – We are excited to share more resources and information with you about how SIRION viral vector services can improve the efficacy, safety, and speed of your gene therapy project.