Alkeus Pharmaceuticals

Clinical-stage biotechnology company developing gildeuretinol (ALK-001) for the treatment of Stargardt disease and other inherited retinal degenerations.

Location
Boston, Massachusetts, USA
Founded
2010
Categories
biotech, rare-disease, ophthalmology, therapeutics

Notes

Alkeus Pharmaceuticals is a clinical-stage biotechnology company focused on developing treatments for Stargardt disease and other forms of inherited retinal degenerations. The company's lead candidate, gildeuretinol (ALK-001), is a modified vitamin A designed to slow the accumulation of toxic vitamin A dimers (A2E) in the retinal pigment epithelium.

Stargardt disease is the most common form of inherited juvenile macular degeneration, affecting approximately 1 in 10,000 people worldwide. ALK-001 has received FDA Orphan Drug and Fast Track designations.

Team

Additional Research Findings

  • In Bain Capital Life Sciences portfolio
  • Lead candidate gildeuretinol (ALK-001) in Phase 3 for Stargardt disease
  • FDA Orphan Drug Designation for ALK-001
  • FDA Fast Track Designation for Stargardt disease
  • Technology based on research from Columbia University
  • Targeting the vitamin A cycle to prevent retinal degeneration
  • Also exploring ALK-001 for dry age-related macular degeneration (AMD)

Sources