ASX listed biotech, Phylogica, has successfully used its first generation cell penetrating peptides, or “CPP’s”, to deliver a protein used for gene editing into the nucleus of a cell line that is normally difficult for genetic material to penetrate.
The successful delivery of the CRISPR/Cas9 protein resulted in a knockdown, or reduction in the expression levels of a receptor on the surface of those cells, due to the gene editing properties of the protein.
Management said the lab testing results establish “proof of concept” for the use of CPPs to deliver CRISPR/Cas9 into target cells.
CRISPR/Cas9 is typically combined with a pre-designed RNA sequence that guides the protein to the right point at the DNA where it then proceeds to cut.
This leads the cell to recognise that the DNA is damaged and it seeks to repair it, allowing scientists to introduce changes to the gene.
CRISPR/Cas9 can potentially be used to treat a range of medical conditions that have a genetic component such as cancer, sickle cell disease and hepatitis B.
Phylogica will now determine the best conditions for CPP assisted delivery of CRISPR/Cas9.
Once this is understood, the company will assess the relative effectiveness and safety of using its CPPs compared to other delivery methods such as electroporation and lipid-based systems.
This testing will be carried out on cells derived directly from a living organism rather than the immortalised laboratory cells used in the initial testing, which will assist with the program’s commercialisation prospects.
In addition, Phylogica has identified and matured second generation CPPs that can deliver up to 10 times as much cargo into the target cell in the CRISPR/Cas9 program compared to the first generation CPPs.
The company has ordered these CPPs and will evaluate them in the first quarter of 2019.
Highlighting the potential of gene-editing, Harvard University recently said that research had started into using CRISPR to change the DNA code inside sperm cells to potentially remove mutations that cause childhood cancer or cystic fibrosis.
By Matt Birney
