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Cell Therapy (Ex-vivo Gene Therapy)

Correcting Defective Genes Ex-vivo to Treat Genetic Disease

Developing lentiviral vectors to genetically modify human cells ex vivo is a common form of gene therapy, and the number of clinical trials using lentiviral technology has rising for many years. Complex lentiviral vector designs for CAR T cells are a prominent example for a successful regulatory approved cell therapy to treat e.g. acute lymphoblastic leukemia (Kymriah, Novartis) and transfusion-dependent β-thalassaemia (Zynteglo, Bluebird).

By serving the entire cell therapy 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 for increased efficacy and patient safety.

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

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

Robust platform processes
1. Manufacturing high quality and high titer preclinical batches 

Inside the Lentiviral Vector Know-How Hub

Technical and Scientific Capabilities of the Lentiviral Vector Hub Development Engine

Development of Drug Substance Blueprints

  • Lentiviral lead vector development
  • Lentiviral vector backbone development
  • Transgene analysis and optimization for efficacious and safe expression profile
  • In-depth project analysis by SIRION cell therapy specialists

Preclinical Manufacturing of Drug Substance Blueprints

  • Lentiviral particle manufacturing
  • High quality manufacturing processes ensure scalable production, optimal productivity and negligible         impurity levels
  • Customizable process development for simple transfer to CDMO Network
  • Extended QC packages compliant to regulatory guidelines

Transfer to CDMO Network and clinical development

  • GMP-ready drug substance blueprint
  • Vector and particle documentation
  • Improve clinical outcome with LentiBOOST® transduction enhancer
  • PoC studies of the lead candidate batches with a CRO network
  • Transfer of Drug Intermediates and processes to GMP network

LentiBOOST® Transduction Enhancer

In lentiviral ex vivo gene therapy, not only the lentiviral particles, but also the cell material and the manufacturing process contribute extensively to the manufacturability and performance of the whole therapy. The recent Mustang Bio Trial MB-207  clearly demonstrated the added benefit of our patented LentiBOOST® transduction enhancer by increasing therapeutic transgene expression and improving clinical outcome.

Contact us – Discover how SIRION can help you reach your gene therapy goals.