Evotec GT

 

Gene therapy is a rapidly evolving modality  enabling curative treatments for a broad range of diseases. This approach involves insertion of nucleic acid into the nuclear genome via a genetically engineered vector.

Evotec’s dedicated gene therapy site, Evotec GT, is located in Austria and provides a  team of experts to cover the full spectrum of services for end-to-end gene therapy development. Our dedicated group of scientists have long-standing experience in applying their research and disease insights to various GT-related technologies and indications. Our comprehensive and combined understanding of the business, product and regulatory landscapes will guide you in designing the optimal strategy for clinical success. 

Our services include the design of state-of-the-art viral AAV vectors for a diverse set of therapeutic payloads, the generation of AAV material for research and non-clinical studies, in vitro and in vivo proof of concept studies for target validation including screening of drug candidates, as well as the design, execution and interpretation of non-clinical gene therapy studies.

With the foundation of Evotec GT our integrated drug discovery platform has further broadened, allowing us to offer gene therapy as new modality that perfectly complements our existing expertise.

In vitro gene therapy

Our discovery portfolio 
  • Selection of suitable AAV capsids with targeted tropism
    • A panel of common wild-type serotype capsids
    • Some off-the-shelf engineered capsids
    • Option to co-develop proprietary own capsid with preferred partners
  • Optimisation of payload for highly efficient vectors
    • Codon optimisation of transgene
    • Protein engineering for improved transgene properties
    • Tailored expression through constitutive or tissue-specific promoter/enhancer elements and other regulatory elements (introns, poly A sequences, posttranscriptional response elements)
    • Expression of therapeutic antibodies 
    • Antisense  approaches to downregulate gene expression (shRNA and miRNA) and for exon skipping (snRNA)
    • Exploration of gene editing concepts
  • Full AAV vector characterisation 
    • Vector quantification and structural characterisation
    • Cell-based in vitro biopotency screening of gene therapy constructs
    • In vitro assays: Cell-based transduction, infectivity and in vitro biopotency
    • Payload specific functional assays
    • Immunological assays
  • CMC
    • Research and non-clinical material (up to 5 L scale)
    • Three plasmid system: Ad helper, Rep-Cap, Payload
    • HEK293 cell lines (suspension & adherent growing)

In vivo pharmacology

Our discovery portfolio
  • In vivo biopotency screening of gene therapy constructs
  • Target biology/pathway: in vivo proof of concept studies
  • Design, execution and interpretation of non-clinical gene therapy studies 
  • Available mouse models or customised disease models
  • Biopotency and pharmocodynamic readouts
  • Combined pharmacology and safety studies
  • Identification of analytical biomarkers and validation in disease models
  • In vivo imaging  
  • Immunohistochemistry and histology
  • Translational pharmacology
  • Vector biodistribution to target and off-target organs
  • Vector shedding
  • Immunogenicity assessment
  • IND-enabling pharm/tox packages

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Our in vivo pharmacology capabilities Our integrated pre-clinical development capabilities

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