Perth-based Lions Eye Institute (LEI) and PYC Therapeutics’ experimental drug for the treatment of retinitis pigmentosa (RP) is now scheduled for human clinical trials in 2021.
The novel drug CPP-ASOs (cell-penetrating peptides antisense oligonucleotides) has successfully corrected the deficiency responsible for one form of RP in ‘retina in a dish’ and phagocytosis models in cells derived from patients with the disease, said LEI’s study co-lead Dr Fred Chen from the University of Western Australia. “This is the best indication that the drug works.”
PYC’s proprietary CPP-delivery platform consists of short chains of amino acids, known as peptides, which due to their sequence and properties can deliver drugs across cell membranes. PYC has successfully demonstrated its CPP-delivery platform in human cells and shown that it outperforms the current gold standard CPP by 400% in a mouse model, said PYC. The CPP delivery system is loaded with ASOs altering the RNA's instructions to the cell to produce healthy, or functional, protein to correct the disease before it presents, it said.
