Evotec GT

Gene Therapy: The future is now

Gene therapy is a rapidly evolving modality enabling curative treatments for a broad range of diseases. Gene therapy aims to treat diseases by replacing, inactivating or introducing genes into cells— either inside the body (in vivo) or outside of the body (ex vivo). Gene therapy is one of the most exciting areas in medicine, both because of recent advances and the opportunities that are emerging.

Evotec GT - Specialised partner for gene therapy development 

Evotec’s dedicated gene therapy site, Evotec GT, is located in Austria and provides a team of experts covering the full spectrum of services for end-to-end gene therapy development. Our dedicated group of scientists has long-standing experience 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 developing the optimal strategy for clinical success. 

Watch the Evotec - Gene Therapy video

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. 

Combining capabilities with our colleagues across Evotec is a great way to speed up the development of new therapies to bring to patients. At the same time, this burgeoning glimpse into a new world of technological possibilities will inspire whole new research areas that will no doubt continue to change the face of biomedical research.

What are we doing at Evotec GT

At Evotec GT, our work includes:

  • Design state-of-the-art viral AAV vectors for a diverse set of therapeutic payloads
  • Generation of AAV and non-viral LNP gene therapy material for research and non-clinical studies
  • In vitro and in vivo studies for target validation, including screening of viral and non-viral gene therapy drug candidates
  • Design, execution, and interpretation of non-clinical gene therapy studies

 Evotec GT - Technical expertise

Technology cornerstones

AAV vector technology
  • Constitutive and tissue-specific promoter/enhancers
  • Ongoing development effort towards proprietary synthetic promoters
  • Common natural capsid serotypes
  • Research licenses for engineered capsids
  • Development of proprietary capsids with the option for co-development partnerships
Non-viral delivery
  • Customisable LNPs for versatile gene delivery
  • Pre-clinical LNP production & characterisation
  • Biodistribution
Payload design
  • Codon-optimised transgenes
  • Vectorised antibodies
  • Antisense approaches (e.g. shRNA, miRNA)
  • Genome editing
Genome editing
  • Selection of suitable editing tools (ZFN, TALEN, CRISPR) and designs, and delivery systems that fit project needs
  • Optimisation of editing components and efficiency in various cellular assays tailored to project needs
  • Integration of in vitro and in vivo areas of expertise
  • Selection of editing and delivery method best suited for the project
  • Design of  guide RNAs and repair templates
  • Assessment of editing efficiency
  • Experimental assessment of the impact of the mutations introduced into the genome
Functional in vitro and in vivo assays
  • In vitro assays: cell-based transduction, infectivity and in vitro biopotency
  • Discovery Sciences
  • Translational pharmacology using humanised and disease models
  • Vector biodistribution to target and off target organs
  • In vivo imaging
  • Combined pharmacology and safety studies
  • Immunogenicity assessment
  • Tox predictions and biomarker identification/validation
  • Integration and off target analysis
CTA/IND-enabling packages
  • Seamless road to clinic
  • IND-enabling pharm/tox packages
  • In vivo release and stability assays
  • Regulatory strategy support

You might also be interested in

Our in vivo pharmacology capabilities Our integrated pre-clinical development capabilities Our "AAV Gene Therapy: Closing the Translational Gap" webinar Our "Evotec and Takeda Gene Therapy" webinar

Contact us


Business Development

T +44.(0)1235.86 15 61 F +44.(0)1235.86 31 39 Get in touch with our experts vCard