gildeuretinol

Gildeuretinol (also known as ALK-001 or gildeuretinol acetate) is a modified vitamin A analog developed as an oral therapy for retinal degenerative diseases, primarily Stargardt disease and geographic atrophy secondary to age-related macular degeneration (AMD). It incorporates a deuterium atom at the CD20 position, which reduces the dimerization of vitamin A—a process implicated in the formation of toxic byproducts that accumulate in the retina due to ABCA4 gene mutations. By slowing this dimerization without modulating the visual cycle, gildeuretinol aims to decrease retinal damage and preserve visual function. Clinical trials have shown that it slows lesion growth and vision loss in Stargardt disease and demonstrates functional benefits in low luminance visual acuity for patients with geographic atrophy. Gildeuretinol has received Breakthrough Therapy, Rare Pediatric Disease, Fast Track, and Orphan Drug designations from the FDA[1][2][3][4][5][6][7][8].

Other names

gildeuretinol acetate