Novel gene therapy vectors

Development of the CRISPR Cas9 gene editing approach has led to an explosion of interest and investment in gene therapy for human health and disease. Most gene therapy approaches use viruses as the delivery vectors for new DNA, but these have severe limitations including: limited packaging sizes, random integration and scaring of the genome, and cell-type dependant expression. 

At BrisSynBio we are developing a novel delivery platform based on an insect virus. We have demonstrated highly effective delivery of large DNA constructs, and are working with major industry partners to bring this technique to the clinic.

Red blood cells as drug delivery agents

The red blood cell (erythrocyte) has a well-characterised life cycle and biophysical properties, making it an ideal vehicle for therapeutic use. This BrisSynBio project will utilize the red blood cell as platform to engineer cells with novel properties for clinical or pharmaceutical applications.

We will do this by engineering changes into in vitro-produced red blood cells, using human peripheral blood stem cells as a starting material. The eventual aim will be to incorporate a range of natural and synthetic proteins into the membrane or cytosol, and thereby introduce novel functionality by influencing cell survival times, drug delivery, enzymatic reactions or storage capacity.

Full details of this project are available here.