Harnessing the Potential of Induced Pluripotent Stem Cells to Accelerate Drug Discovery for Age-Related Macular Degeneration

The poster Harnessing the Potential of Induced Pluripotent Stem Cells to Accelerate Drug Discovery for Age-Related Macular Degeneration presents Axol Bioscience’s development of a scalable, genetically stratified in vitro model for dry AMD using patient-derived iPSC-RPE cells.

Key highlights include:

• Model Design:
  • RPE cells derived from multiple AMD patient iPSC lines
  • Chronic A2E exposure followed by blue light simulates lipofuscin accumulation and oxidative stress
• Functional Validation:
  • PCi-RPE cells express MITF, PMEL17, ZO-1
  • Demonstrated phagocytosis capability via pH-Rhodo assay
• AMD Hallmarks Recapitulated:
  • Increased oxidative stress (CellROX)
  • Complement activation (C3 staining)
  • Elevated IL-6 and IL-8 secretion (ELISA)
  • Upregulation of drusen-related genes (APOE, APOJ)
  • RPE atrophy confirmed via live/dead assays
• Genetic Stratification:
  • 26 patient-derived iPSC lines with CFH, HTRA1, ARMS2 risk alleles
  • Risk allele carriers show increased sensitivity to A2E + blue light stress
  • Enables “clinical trial in a dish” approach
• Future Directions:
  • Integration of photoreceptors and microglia into the AMD model
  • Expanded screening of therapeutic compounds

This model supports personalized AMD research and drug screening with high physiological relevance.

Featured Axol Products:

• PCi-RPE Cells (iPSC-Derived Retinal Pigment Epithelium) – PCi-RPE
• PCi-RPE Differentiation & Maturation Protocols – (internal Axol protocols)
• PCi-RPE AMD Model Assay Kit – (includes A2E treatment and blue light exposure workflow)