Lysoclear, Inc. is a pre-clinical Ichor Therapeutics portfolio company that develops enzyme therapies for eye disease. The company is currently developing leads to treat atrophic “dry” age-related macular degeneration and Stargardt’s macular degeneration.
Age-related macular degeneration (AMD) and Stargardt’s macular degeneration (SMD) are thought to arise from the gradual loss of retinal pigmented epithelial (RPE) cells of the macula, the area of the eye responsible for central vision. The accumulation of toxic vitamin A aggregates have been implicated in these diseases.
Recent research suggests that vitamin A aggregates are capable of binding native lysosomal enzymes, inhibiting their function. As the accumulation of these aggregates reaches a critical threshold, lysosomal impairment leads to the accumulation of intracellular lipofuscin, extracellular drusen deposition, and eventually RPE cell death.
Lysoclear is a recombinant enzyme product under development by Ichor Therapeutics that is able to selectively localize to the lysosomes of RPE cells where toxic vitamin A aggregates accumulate, and destroy them.
Lysosomal storage diseases (LSDs) are a class of rare congenital illnesses that result from defects in endogenous lysosomal enzymes. Many LSDs are effectively treated in the clinic with recombinant forms of the defective enzyme. When “decorated” with mannose sugars, the recombinant enzyme is efficiently shuttled to the lysosomes of target cells, restoring function.
Lysoclear operates identically to existing LSD therapies. However, instead of replacing a defective enzyme, a new enzyme is introduced that upgrades RPE cells with the ability to degrade A2E and other lipofuscin components.