Skip to main content

International 3Rs Prize now open for applications. £30k prize (£2k personal award) for outstanding science with demonstrable 3Rs impacts.

NC3Rs | 20 Years: Pioneering Better Science
Online

10 years of CRACK IT webinar: Assessing the genotoxic potential of gene therapy products in vitro

-

Registration Details

Event date and time
-
Organiser
NC3Rs
Location
Online
CRACK IT 10: ten years of innovation

This webinar forms part of the CRACK IT 10 year anniversary celebrations. It focuses on the development and launch of TestAVec, a company that in collaboration with Challenge partner GeneWerk GmbH has developed the hInGeTox - Human In Vitro Gene Therapy Genotoxicity Testing service. TestAVec was founded on technology developed to address the 2015 InMutagene CRACK IT Challenge sponsored by GlaxoSmithKline (GSK) and Novartis. 

Register via Zoom

The TestAVec team, in collaboration with lead contractor GeneWerk GmbH, has developed a human stem cell based in vitro testing service (hInGeTox) to reduce the use of animals in assessing the genotoxic risk of gene therapy products.​ These products use therapeutic DNA sequences to replace or disrupt existing genes and are delivered using vector systems which can themselves cause genotoxicity. hInGeTox assesses the safety and efficacy of vectors early in development using human induced pluripotent stem cells that have been differentiated to hepatocytes and cultured in 3D. Several vector-related factors that cause genotoxicity can be measured using hInGeTox. These include effects on cancer gene expression by vector promoters and enhancers, aberrant transcript forms or gene splicing, host epigenetic responses to gene transfer and therapeutic gene expression. The team is also exploring the use of patient-derived cells in these assays to provide a personalised medicine approach.

During this webinar, which is free to attend, you will hear from one of the developers of hInGeTox and from the challenge Sponsor. There will be an opportunity to ask questions after the webinar presentation.

Register via Zoom

Speakers 

  • Dr Mike Themis, Founder and Chief Scientific Officer of TestAVec, the company spun out to commercialise the research developed during the InMutagene Challenge. Mike will describe the technology developed and the partnership they have established with GeneWerk GmbH to offer a personalised genotoxicity testing service.

  • Dr Rhiannon Lowe (GSK). Rhiannon has worked with TestaVec and Genewerk GmbH as one of the Sponsors of the InMutagene Challenge. She will describe the background to the Challenge, the role of the Sponsor and how GSK provided in-kind support through a fully funded studentship. Rhiannon will also describe the scientific and 3Rs benefits that services from TestaVec and GeneWerk GmBH provide to the growing areas of cell and gene therapy.


Background 

The InMutagene CRACK IT Challenge, sponsored by GSK and Novartis and launched in 2015, aimed to develop in vitro and/or in silico assays that can be used singly or in combination to improve risk assessment for gene therapy products. In the development of gene therapies, there is an increasing demand for preclinical tests to assess the safety of vectors used to deliver therapeutic DNA sequences. Existing models to test vector-mediated genotoxicity often use animals or animal-derived cell lines. While offering readouts for transformation and other markers of genotoxicity, they can be unreliable or overestimate the risk of genotoxicity. Alongside this, preclinical models of oncogenesis are expensive and time-consuming, and the long-term duration of studies can present ethical concerns. 

Further information about the TestaVec platform and InMutagene Challenge can be found on the NC3Rs Innovation Platform. 


Downloads