Focus Area Advanced Therapeutics

Cell Therapies for Ophthalmology

The Client

  • Large Japanese Pharmaceutical company heavily dependent on three marketed products for immunology, genitourinary, and oncology indications
  • Additional therapeutic areas of interest include cardiology, diabetes, gastrointestinal disease, infectious disease, neurology and ophthalmology


  • Assess the potential for regenerative cell therapies for dry AMD with geographic atrophy, diabetic retinopathy, glaucoma and inherited retinal diseases (e.g., retinitis pigmentosa)
  • To help the client determine which of four different cell therapy programs should be prioritized for development


  • Secondary research to better understand epidemiology, diagnosis, segmentation and management of patients with each indication of interest
  • Primary research with KOL-level ophthalmologists to assess the potential of cell-based regenerative therapies for each indication of interest

Valued Outcome

  • Overviews of each indication of interest focusing on relative potential for regenerative cell therapy and target product profile required to support approval and uptake
  • Recommendations regarding the specific cell therapy program and indication with the greatest clinical and commercial potential