Project: Lipofuscin-Destroying Enzymes to Treat Macular Degeneration

Lysosomes are the cell's waste incinerators, responsible for destroying all kinds of molecules when they are no longer needed. Thus, they harbor an impressive array of enzymes capable of attacking and breaking many substances, but not all. Some molecules are formed so slowly that evolution "did not select for" an enzyme for degrading them. Rather, these molecules are stored in the lysosomes, and accumulate over the entire life span until they get in the way of the affected cells' normal functioning and cause disease. Medical bioremediation is the proposal to destroy these molecules using lysosomal enzyme therapy. See De Grey AD, Alvarez PJ, Brady RO, Cuervo AM, Jerome WG, McCarty PL, Nixon RA, Rittmann BE, Sparrow JR. Medical bioremediation: prospects for the application of microbial catabolic diversity to aging and several major age-related diseases. Ageing Res Rev. 2005 Aug;4(3):315-38. PMID 16040282.

The target

One key target for medical bioremediation is A2E, a by-product of the photochemistry of vision. A2E accumulation is thought to cause Stargardt's Macular Degeneration, and is suspected to be involved in age-related macular degeneration. There is currently no known means to remove A2E safely. Current treatments for macular degeneration are aimed at suppressing symptoms, rather than addressing underlying cause, and include approaches such as squinting around the degenerating dark spot in the visual field, and wearing blue glasses to limit photodamage. There are no cures or disease-modifying treatments approved for any form of macular degeneration.

The intervention

We have discovered a panel of naturally occurring enzymes which destroy A2E. These are the only such enzymes known. Currently, we are working on a drug delivery system to get these enzymes into the affected retinal pigment epithelial cells' lysosomes. A patent on these enzymes is pending, and is expected to be assigned jointly to SENS Foundation and the Arizona State University, our collaborator in this research.

Licensing

The RC carries out its work in order to further the mission of SENS Foundation, which includes ensuring widespread access to the regenerative medicine solutions which we develop. We choose partners who share this long-term vision, and can provide the resources to pursue its realization.

In the case of our work on macular degeneration we are looking for licensing partners with particular attributes:

• no significant stake in competing products,
• a track record of outstanding management and solid financing,
• resources to support, and expertise in, protein drug development,
• a pre-clinical ophthalmological program, or connections to ophthalmology CROs,
• experience in delivering therapeutics to the lysosome.

If you are interested in this opportunity please contact us.