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Perth partnership targets childhood blindness and $1 billion market

The Lions Eye Institute (LEI) has signed a commercial partnership that will combine its antisense gene therapy for a leading cause of childhood blindness with a new Australian-developed drug delivery platform.

LEI and ASX-listed PYC Therapeutics have entered into a commercial collaboration agreement that will initially develop drugs to treat retinitis pigmentosa. The partnership sees the formation of a new Perth-based special purpose organisation called Vision Pharma, to which LEI and PYC have contributed equal levels of intellectual property.

Vision Pharma will combine PYC’s cell penetrating peptide (CPP) drug delivery technology with a proprietary LEI drug known as an antisense oligonucleotide (ASO). The ASO has already shown preliminary evidence of successfully reversing the effects of a genetic mutation in human cells associated with retinitis pigmentosa.

With no existing treatment options available for the disease, it is thought the therapy’s potential target market could top $1 billion per year.

The drug is currently in advanced pre-clinical development and is scheduled to enter investigational new drug-enabling studies during the first half of 2020. If successful, it will be approved for testing in humans.

“LEI is proud to see one of its research developments now entering pre-clinical trials to treat the commonest form of retinitis pigmentosa,” LEI managing director Professor Bill Morgan said.

“Professor Fred Chen (LEI) and Professor Sue Fletcher (formerly of Murdoch University) have been working on their genetic therapy and are partnering with PYC, using their transporter protein to deliver this novel therapy directly to the sick retinal cells. We are very excited about the possibility of being able to cure this form of blindness.”

Chen, co-inventor of the lead drug candidate and head of LEI’s Ocular Tissue Engineering Laboratory, said the agreement was the culmination of decades of splice therapy research. Now, this work is being combined with PYC’s CPPs, data from the Australian Inherited Retinal Diseases Registry and LEI’s expertise in stem cell retinal disease modelling.

“The advantage of using ASO is that it’s personalised medicine; specific for the gene or the mutation. Its delivery into the cell does not require the introduction of a viral vector into the eye and into the retinal cell where the transferred gene is not under the normal regulation, resulting in the potential for over or under production of the protein coded by the gene.

“ASO treatment is different from gene transfer therapy; it manipulates the way in which gene is processed and expressed a level downstream from the DNA – at the RNA level – where splicing occurs.

“We estimate that a third of the patients with inherited retinal disease may carry splice altering mutations that might be treatable with this method.”

According to PYC, its drug delivery technology addresses a major obstacle in precision medicine by being able to deliver precise and potent drugs to targets inside cells.

Vision Pharma is also developing a pipeline of other ASO candidates for the treatment of different forms of inherited retinal disease and expects to progress them into the clinic alongside the lead drug candidate.

PYC also announced that Fletcher, a global leader in the design and development of RNA therapeutics, has been appointed as chief of research and development on a part-time basis. She will be tasked with supporting both the retinitis pigmentosa program and building PYC’s pipeline of CPP-ASO drugs.