Development of chiral fluorinated alkyl derivatives of emixustat as drug candidates for the treatment of retinal degenerative diseases

Eliav Blum, Jianye Zhang, Edward Korshin, Krzysztof Palczewski, Arie Gruzman

Research output: Contribution to journalArticlepeer-review

Abstract

The discovery of how a photon is converted into a chemical signal is one of the most important achievements in the field of vision. A key molecule in this process is the visual chromophore retinal. Several eye diseases are attributed to the abnormal metabolism of retinal in the retina and the retinal pigment epithelium. Also, the accumulation of two toxic retinal derivatives, N-retinylidene-N-retinylethanolamine and the retinal dimer, can damage the retina leading to blindness. RPE65 (Retinal pigment epithelium-specific 65 kDa protein) is one of the central enzymes that regulates the metabolism of retinal and the formation of its toxic metabolites. Its inhibition might decrease the rate of the retina's degeneration by limiting the amount of retinal and its toxic byproducts. Two RPE65 inhibitors, (R)-emixustat and (R)-MB001, were recently developed for this purpose.

Original languageEnglish
Article number127421
JournalBioorganic and Medicinal Chemistry Letters
Volume30
Issue number18
DOIs
StatePublished - 15 Sep 2020

Bibliographical note

Publisher Copyright:
© 2020 Elsevier Ltd

Keywords

  • (R)-MB001
  • (R)-emixustat
  • Fluorinated alkyles
  • RPE65 (Retinal pigment epithelium-specific 65 kDa protein
  • Retinal
  • Retinal degeneration

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