Cell Penetrating Peptides and PepGen Limited (2018)
Dr Mike Gait and his team together with Professor Matthew Wood’s laboratory at the University of Oxford have collaborated for over 10 years to develop a series of novel cell penetrating peptides (CPP).
Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Dystrophy are genetic disorders characterised by progressive muscle degeneration and weakness. Modulation of pre-mRNA splicing using splice-switching oligonucleotides is a promising approach to restore the expression of the proteins. It is their absence that causes these disorders. Currently though there is no efficient delivery system to allow sufficient levels of oligonucleotides reaching the pre-mRNA target. CPPs have been developed to overcome this. The first generation of CPP developed were the “Pip” CPP family based on Penetratin. Gait and Wood’s teams later developed “bispecific” CPPs which allow delivery of two oligonucleotides using a single CPP. A patent application was filed in 2015. There are also now DPEP CPPs that represent a further improvement in activity whilst reducing toxic side effects. A patent application claiming DPEP CPPs was filed in 2017.
PepGen, is a new company which spun out in early 2018 and will take forward the development and commercialisation of the “bispecific” CPPs and the DPEP CPPs.