In 2017, Ichor Therapeutics announced the launch of LYSOCLEAR, a therapeutic approach that proposes to reverse age-related macular degeneration and the related Stargardt macular degeneration.
Its line of research is based on finding naturally occurring bacterial enzymes that are able to break down metabolic waste with which our own cellular machinery struggles or cannot cope, particularly the waste product lipofuscin. Lipofuscin and its compounds vary in type from tissue to tissue, and they accumulate in the lysosome of the cell, a cellular recycling plant that breaks down waste.
Unfortunately, our lysosomes cannot cope with lipofuscin and are unable to fully break it down; over time, this leads to the lysosomes becoming clogged with insoluble garbage that eventually stops them from working. The end result is that every cell in our body becomes increasingly dysfunctional as lysosomal activity declines and the garbage levels rise.
In general, the exact link between lipofuscin accumulation and the development of age-related diseases is not fully understood, but, in some diseases, the role of lipofuscin in disease progression is clear. This is the case for age-related macular degeneration and is why Ichor focuses on the compound N-retinylidene-N-retinylethanolamine (A2E), a component of ocular lipofuscin.
LYSOCLEAR is an enzyme product that can enter the lysosomes of retinal pigmented epithelial (RPE) cells, where A2E accumulates, and destroy it. Ongoing studies suggest that LYSOCLEAR is safe and effective at targeting A2E, eliminating up to 10% of it with each dose. An Investigational New Drug application and Phase 1 clinical trials are slated for 2018, and they aim to test this new therapy in humans to identify the optimal dosages and regimen of application.