February 15th, 2017
LAFAYETTE, NY: Today, Ichor Therapeutics, a biotechnology company that focuses on developing drugs for age-related diseases, announced a series A offering to bring its LYSOCLEAR product for age-related macular degeneration and Stargardt’s macular degeneration through Phase I clinical trials. This product would be the first clinical candidate based on the SENS paradigm, pioneered by biomedical gerontologist Dr. Aubrey de Grey.
Age-related macular degeneration (AMD) is the leading cause of vision loss among people over the age of 50. Over 20 million Americans are affected by this condition. There are two forms of AMD. Atrophic or “dry” AMD represents 85% of all cases, and arises from the progressive loss of photoreceptors in the macula – a part of the eye responsible for central vision. A juvenile form of macular degeneration called Stargardt’s macular degeneration (SMD) also exists, and is caused by mutations in the ABCA4 gene, which plays an essential role in vitamin A metabolism.
In later stages of disease, dry AMD can progress to a more advanced “wet” form, accounting for the remaining 15% of all AMD cases. Wet AMD is characterized by neovascularization (new blood vessel formation). Exudate and hemorrhage can further accelerate vision loss and lead to complete blindness.
There is no cure for dry AMD or SMD. The AREDS vitamin formulation and cessation of smoking have been shown to mildly slow progression. Wet AMD is modestly managed with monthly injections of antibodies that inhibit the vascular endothelial growth factor (VEGF) pathway.
The underlying pathology of AMD is thought to be caused by the death of retinal pigmented epithelial (RPE) cells, which photoreceptors in the macula rely upon to survive. RPE cells assist photoreceptors in various metabolic roles, including the recycling of vitamin A, an essential component of the visual cycle. However, this is a leaky process, and trace by-products are formed that accumulate in the lysosomes of RPE cells. The most well studied of these by-products is A2E, a toxic compound which may play a causative role in AMD and SMD.
Although A2E accumulates gradually over the lifespan, it is generally not until later age that A2E reaches a threshold necessary to promote toxicity. At high concentrations, A2E promotes the formation of intracellular junk termed lipofuscin. RPE cells attempt to handle this accumulation by shuttling the junk out in the form of extracellular drusen. Eventually, the RPE choke on the garbage, and cell death accompanies complement activation, inflammation, and hypoxia.
Multiple companies have developed drugs that successfully reduce the rate of A2E formation, but such interventions may be too late for symptomatic patients, who have already had the cascade kicked off.
LYSOCLEAR: the junk buster
In 2014, Ichor Therapeutics completed a material and technology transfer agreement for rights to concepts and research pioneered by SENS Research Foundation. In 2017 Ichor announced LYSOCLEAR, a recombinant enzyme product that selectively localizes to the lysosomes of RPE cells where A2E accumulates, and destroys it. Ongoing studies suggest that LYSOCLEAR is safe and effective at targeting A2E, eliminating up to 10% with each dose.
Ichor is a shining example of our mission to kick-start the rejuvenation biotechnology industry with a project that we initiated with early donor support. Led by one of our most dynamic and accomplished alumni, Kelsey Moody, Ichor is forging ahead faster than we could have dreamed.
– Aubrey de Grey
LYSOCLEAR is under continued development by Ichor Therapeutics and could bring much needed relief to millions of Americans afflicted by AMD and SMD.
Ichor has opened a Series A funding round to support pre-clinical Investigational New Drug (IND) enabling studies and phase I human clinical trials for AMD and SMD. The company has established a partnership with Syracuse University and obtained dedicated seed funding for the program from Michael Greve´s Kizoo Technology Ventures, SENS Research Foundation, FightAging.org, CenterState CEO, and several private investors to support its present work on LYSOCLEAR.