PYC Therapeutics, (ASX:PYC) has reported promising results from its exploration of the potential of a novel non-viral delivery technology for gene therapy.
The company is investigating various methods for intra cellular delivery of a range of high value drug cargoes that can’t be covalently linked to its Cell Penetrating Peptides (CPPs).
The cargoes of interest include siRNA, mRNA, DNAzymes and DNA therapies. Positive results would open up a new paradigm of drug delivery by overcoming the safety and production concerns associated with delivering these drug cargoes using engineered viruses (eg. Adeno-Associated Virus or AAV platforms).
Sue Fletcher, PYC’s CSO, says that despite the enormous interest in gene therapy, the field has been held back by the lack of safe and effective genetic drug delivery technologies. The currently utilised viral approaches are restricted by uneven distribution between cells and a raft of safety concerns. Non-viral approaches are therefore preferred, yet suffer from low drug uptake (transfection) in target cells (efficacy). Lipid NanoParticles (LNPs) are a leading form of non-viral delivery technology for gene therapies that are increasingly being adopted in clinical development programs (including by Moderna’s mRNA vaccine for COVID-19).
According to Ms Fletcher, improved transfection efficiency (efficacy) is the key to broader uptake of LNPs as a delivery technology for gene therapy. The results described below demonstrate the utility of combining PYC’s proprietary CPPs with LNPs to improve their transfection efficiency (efficacy).
In vitro data evaluating PYC’s CPPs embedded in the surface of Lipid NanoParticles (LNPs) demonstrate a substantial improvement in efficacy of the LNPs with no observed toxicity – opening up a path to non-viral DNA and RNA drug delivery.
The LNPs used in these experiments were developed in the laboratory of Professor Dan Peer, from Tel Aviv University (TAU), patented by Ramot, the technology transfer company of TAU and funded by The Technology Innovation Momentum Fund.
Professor Dan Peer is a leading expert in the field of LNPs whose technology is currently in clinical development and who has published widely on the subject matter.
The next step for PYC is to demonstrate the utility of these CPP-LNP combinations in animal models.
Our CPP-LNP delivery technology is an exciting development that we expect will open the door to many additional nucleic acid drugs,” Ms Fletcher said
“We are particularly delighted by this demonstration of the benefits of our collaborative approach to research and drug development and thank Prof. Dan Peer for joining us on this journey.”