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Gildeuretinol

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Pharmaceutical compound
Gildeuretinol
Clinical data
Other namesALK-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
PubChem CID
UNII
KEGG
ChEMBL
Chemical and physical data
FormulaC20H30O
Molar mass286.459 g·mol
3D model (JSmol)
SMILES
  • C()()/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 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 a deuterated derivative of Vitamin A that is designed to reduce the dimerization of vitamin A without affecting the visual cycle.

Gildeuretinol has received breakthrough therapy and orphan drug designations from the U.S. Food and Drug Administration.

References

  1. Zaydon YA, Tsang SH (July 2024). "The ABCs of Stargardt disease: the latest advances in precision medicine". Cell & Bioscience. 14 (1): 98. doi:10.1186/s13578-024-01272-y. PMC 11282698. PMID 39060921.
  2. Fitch J (22 November 2024). "Gildeuretinol for Stargardt disease receives Rare Pediatric Disease, Fast Track Designations". Contemporary Pediatrics.
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