I believe that the developer of the gene therapy for dry AMD which is being trialled in Melbourne is Gyroscope Therapeutics. They are targeting the geographic atrophy which is secondary to dry AMD.
Their theory is that an overactive complement system (which is part of the immune system) causes inflammation that damages healthy eye tissue, leading to vision loss. Using gene therapy, they aim to activate production of more Complement Factor 1 protein to naturally dampen the overactivity of the complement system.
The geographic atrophy secondary to dry AMD can also be targeted using an antisense approach. Ionis is currently running a Phase 2 trial to assess safety and efficacy of multiple doses of IONIS-FB-LRx, an antisense inhibitor of Complement Factor B, in patients with geographic atrophy secondary to Age-related Macular Degeneration (AMD).
It’s estimated that the 330 patient Phase 2 trial will last three and a half years and complete in October next year. If successful, I would expect that there would be two further multi-year Phase 3 trials required, each with ~1,000 patients.
If you look at PYC’s June 2020 presentation, Slide 24, it seems to be suggested that there is potential use for PYC’s CCP-PMO drug delivery platform in both dry and wet AMD.
However, PYC has opted instead to pursue rare inherited retinal diseases for its leading ocular programs. There are a number of reasons for this – clearly defined targets, smaller and fewer trials required, much lower cost, faster time to market, favourable FDA regulatory pathways, extended market exclusivity and a less crowded competitive landscape.
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