OpRegen® is currently being evaluated in a Phase 1/2a open-label, dose escalation safety and efficacy study of a single injection of human retinal pigment epithelium cells (RPE cells) derived from an established pluripotent cell line and transplanted subretinally in patients with advanced dry age-related macular degeneration (AMD) with geographic atrophy (GA). In November 2020, Lineage announced the completion of patient enrollment in this study.

The FDA has granted OpRegen with Fast Track Designations and, as such, is on an expedited regulatory path that includes the ability for increased interfacing with the FDA during clinical development and enhanced favorability for marketing approval.

Clinical Data

In November 2021, Lineage reported that restoration of retinal tissue was observed in a fourth patient enrolled in the Company’s Phase 1/2a clinical study of OpRegen.

  • Four patients with dry age-related macular degeneration observed to have areas of geographic atrophy which diminished or remained unchanged relative to baseline for a period of at least 12 months
  • All four patients exhibited improvements in visual acuity at 12 months
  • Differences in visual acuity between treated and untreated eyes in all cohort 4 patients remained statistically significant at 9, 12, and 15 months
Dry AMD RPE degeneration and drusen accumulation

Image adapted from scienceofamd.org

Market Opportunity

AMD affects more than 30 million people worldwide and approximately 1.6 million people are newly diagnosed annually in the U.S.1 It is a leading cause of vision loss in people over the age of 60 in the developed world. There are two forms of AMD: “wet AMD,” which affects only 10% of patients and “dry AMD,” which affects 90% of patients.2 Currently, there are only two FDA-approved therapies for the less common wet AMD, yet they constitute an estimated market of more than $10 billion.3

There are no FDA approved medical therapies for the 90 percent of AMD patients who suffer from the dry form. We believe one of the most promising future therapies for dry AMD is the replacement of the layer of damaged RPE cells that support and nourish the retina.

AMD Market

Sources: (1) Pennington and DeAngelis, Eye and Vision, 2016 3:34; (2) JM Seddon, Epidemiology of age-related macular degeneration. (AP Schachat, S Ryan eds.) Retina, 3rd ed. St. Louis, MO: Mosby; 2001;1039-50 and (3) 2018 product sales summary based on publicly reported revenue figures for Lucentis and Eylea.

View our pipeline to see our other programs in development.