AAV Vectors for Gene Therapy
Supporting Gene Therapy Development with Safer, More Efficacious AAV Vectors
Recombinant AAV vectors dominate the in vivo gene therapy field aiming to treat rare genetic diseases. There are already several licensed gene therapies for this small and mighty vector and many more are expected to follow.
Improving the proportion of AAV gene therapy trials meeting their primary efficacy and safety endpoints is a continuous endeavor. It is SIRION Biotech’s mission to enable CGT developers to develop tomorrow’s gene therapies with the right tools, technologies and expertise in a field that is evolving quickly from a scientific and a regulatory perspective.
SIRION Biotech provides a 360° end-to-end solution within the R&D and preclinical rAAV space to develop safe and efficacious rAAV vector drug products.
SIRION supports the development of all three key elements in rAAV drug development for clinical application:
SIRION Key Advantages
SIRION Biotech can de-risk your gene therapy development programs by sharing 12 years of experience in viral vector development plus in-depth knowledge of market trends via working with our extensive client base active in CGT.
At SIRION Biotech, the development of all rAAV gene therapy key elements can be integrated into one cohesive lead vector development program with a dedicated SIRION project manager, ensuring seamless interplay of all developed elements, reduction of development time, and minimizing project management time for our partners.
Needs & Technology Focused
SIRION dedicates all tools and technologies to our CGT development partners and clients and does not develop drugs for an internal pipeline. We create custom lead vector development programs focused on client needs, including a scientific expose, experimental strategy, and roadmap. Clients can choose to work with us on any or all of the 3 key development elements according to specific needs. We are constantly updating and expanding our technological portfolio according to the latest scientific insights and are also excited to develop new assets in collaboration with our GT clients.
SIRION Biotech rAAV Vector Development Capabilities
Therapeutic Expression Cassette Optimization
- Therapeutic transgene expression cassette optimization for improved efficacy and safety including high-through put expression cassette screening
AAV Capsid Selection and Engineering
- AAV capsid evolution in IP generating partnerships
- Nanobody redirected capsid targeting in IP generating partnerships
- Vector biodistribution studies (on target vs. off-target organs)
Manufacturing technologies, process development and tech transfer
- Proprietary manufacturing processes optimized for minimal process - and product related impurities for research and preclinical studies
- Manufacturing tools and technologies for improved cost efficiency, safety and productivity such as rAAV vector backbones optimized for clinical application
- In-house process development to overcome challenges with custom solution and GMP-transferrable processes
- Documentation of plasmids, AAV vectors and manufacturing process to serve as basic material for the GMP manufacturing of the AAV vectors and support IND filing
Manufacturing for Preclinical Studies
Manufacturing of AAV vectors for research and preclinical studies follows our ISO certified manufacturing procedure. Our experienced process development team also establishes advanced manufacturing procedures on demand for individual requirements.
Our manufacturing processes are designed to minimize:
process related impurities (e.g. transfection, cell medium, host cell derived or purification derived)
product related impurities (empty capsids or incomplete packaged AAV vector genomes)
We offer a broad range of extended QCs to monitor and document purity, potency, safety, and identity of the AAV plasmid backbone, therapeutic transgene cassette, and resulting AAV vectors. The manufacturing procedures, QC analytics, and documentation are specific to the project and tailored to the exact requirements of each CGT developer.
Our preclinical manufacturing processes – including custom developed advanced manufacturing procedures – are transferrable to partners within our closely aligned, worldwide CDMO network for GMP-manufacturng. This significantly speeds up the transfer process. We can additionally transfer the plasmid sequences for AAV drug substance manufacturing and provide physical plasmid starting material via plasmid CDMO partners.