This technology is a structural class of transthyretin (TTR) ligands that can be used to simultaneously treat dry age-related macular degeneration (AMD) and wild-type transthyretin amyloidosis (ATTRwt).
Age-related macular degeneration (AMD) is the most common cause of blindness in developed countries, particularly in people older than 60 years. Wild-type transthyretin amyloidosis (ATTRwt) is a late-onset non-genetic disease associated with aggregation of wild-type transthyretin (TTR). There is no treatment for the most prevalent dry form of AMD. While retinol-binding protein 4 (RBP4) antagonists can be an optimal treatment option for most dry AMD patients, this class of drugs can be counter-indicated for a subset of individuals with genetic or age-related predisposition to TTR amyloidosis. There is a significant medical need for a treatment for dry AMD without complications due to ATTRwt.
This technology is a structural class of transthyretin ligands capable of dissociating the RBP4-TTR complex to partially reduce the retinol traffic to the retina and inhibit the formation of lipofuscin bisretinoids. A TTR tetramer released from the RBP4-TTR complex is then stabilized with the same ligand thus preventing its potential aggregation. This class of TTR ligands may be effective at treating a host of macular degeneration diseases as well as TTR amyloidosis-based diseases. The technology may be used to treat uncomplicated forms of macular degeneration (such as dry AMD and Stargardt disease), as well as different genetic or sporadic forms of TTR amyloidosis not associated with macular degeneration.
Patent Pending (US20240150297)
IR CU20308
Licensing Contact: Kristin Neuman