Gildeuretinol

Gildeuretinol
Clinical data
Other names	ALK-001, KL-49
Identifiers
	IUPAC name (2E,4E,6E,8E)-7-methyl-3-(trideuteriomethyl)-9-(2,6,6-trimethylcyclohexen-1-yl)nona-2,4,6,8-tetraen-1-ol;
CAS Number	118139-35-8;
PubChem CID	169490774;
UNII	PSZ7W5NR24;
KEGG	D12713;
ChEMBL	ChEMBL5314606;
Chemical and physical data
Formula	C20H30D3O
Molar mass	292.500 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES [2H]C([2H])([2H])/C(=C\CO)/C=C/C=C(\C)/C=C/C1=C(CCCC1(C)C)C;
	InChI InChI=InChI=1S/C20H30O/c1-16(8-6-9-17(2)13-15-21)11-12-19-18(3)10-7-14-20(19,4)5/h6,8-9,11-13,21H,7,10,14-15H2,1-5H3/b9-6+,12-11+,16-8+,17-13+/i2D3; Key:FPIPGXGPPPQFEQ-QZNDTXPVSA-N;

Gildeuretinol is an investigational new drug being developed by Alkeus Pharmaceuticals, Inc. for the treatment of retinal diseases, particularly Stargardt disease and geographic atrophy secondary to age-related macular degeneration (AMD). Stargardt disease is caused by a defect in the ABCA4 gene that clears toxic byproducts resulting from the dimerization of vitamin A. Gildeuretinol is new molecular entity designed to reduce the dimerization of vitamin A in the eye without affecting the visual cycle.^[1]

Gildeuretinol has received breakthrough therapy, orphan drug and Pediatric Rare Disease designations from the U.S. Food and Drug Administration.^[2]

References