MCW to use Transposagen's 'more flexible than ZFN' gene editing tools

Transposagen Biopharmaceuticals is providing its gene editing tools to the Medical College of Wisconsin (MCW) as part of an $8.7m (€5m) grant-funded project to create new genetically modified rat models for studying the genetics of human diseases.

“MCW will provide Transposagen with gene target information for their projects,”​ Transposagen’s Director of Sales and Marketing Jack Crawford told Biopharma-Reporter.com, and in return “Transposagen will design and produce gene editing tools for those targets and send them to MCW to use in-house to create genetically engineered rats.”​

Gene Editing Nucleases​

In this agreement, MCW will be using XTN Talens which, Crawford said, “are DNA binding proteins that can be designed to specifically bind to any site in the genome. Once it is bound to its target the XTN Talen will cut the DNA at that specific site resulting in genetically engineered cells and animals.”​

The technology is more flexible than alternative ZFN gene editing tech as it allows more locations in the genomes to be accessible for gene editing than with ZFN, he said. For example, one of the firm’s pharmaceutical partners recently found success with Transposagen’s platform after years of failed attempts using ZFNs from other providers.

“These tools are the best out there for drug target discovery and creation of cell and animal models for drug development,”​ said Crawford. Furthermore: “The tools we produce are also very useful for creating genetically engineered cell lines for bioprocessing and protein/antibody production.”​

piggyBac​

Transposagen also holds exclusive licenses and patents to the piggyBac Gene Modification System, which can be used in combination with site-specific nucleases for gene editing in cells and animals, whilst avoiding the problem of leaving unwanted pieces of DNA that may alter or silence the gene expression and leads to less useful experiences and assays.

PiggyBac “is capable of integrating exogenous DNA into the genome but more importantly being able to remove it from the genome without leaving behind any mutations,”​ said Crawford. “This system allows pharma companies to select cell lines for a particular gene change and then remove the ‘selectable marker’ (usually a drug resistance gene or fluorescent gene) without leaving any mutations in the genome other than the ones they wish to make.”​

As for how Transposagen’s technologies are being used, Crawford told us whilst the Talen tech is focused on cell line engineering, the combination of XTN and piggyBac platform – known as Footprint-Free Gene Editing - is the most suitable for stem cell engineering.

“In addition to research animal and cell line engineering our technologies have also demonstrated utility for plant gene modification and we have demonstrated this for a commercial project in tobacco as well as agricultural animal modification.”​