Key Takeaways

  • Three-year data show sustained visual acuity gains and retinal structural improvements following a single administration of OpRegen in GA patients
  • Greater benefits were observed in patients with more extensive treatment coverage, with evidence of partial retinal restoration and photoreceptor recovery
  • Ongoing phase 2a GAlette study aims to optimize delivery techniques and further evaluate the therapy’s potential to modify disease progression

Lineage Cell Therapeutics announced new 36-month data from its phase 1/2a clinical study of RG6501 (OpRegen), an investigational retinal pigment epithelium (RPE) cell therapy for geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The results were presented at the Foundation Fighting Blindness Retinal Therapeutics Innovation Summit 2026.

The presentation, delivered by Eyal Banin, MD, PhD, on behalf of Roche and Genentech, highlighted sustained visual and anatomical benefits following a single subretinal administration of OpRegen in select patients.

“We are excited that our partners continue to collect data and highlight positive clinical results from the OpRegen RPE cell therapy phase 1/2a program,” said Brian M. Culley, CEO of Lineage. “Long-term clinical outcomes from RPE cell therapy are challenging the long-held view that GA is an irreversible condition.”

Retinal Innovation Summit 2026 Highlights

  • Gains in Best Corrected Visual Acuity (BCVA) in patients in Cohort 4 (less advanced GA than in other cohorts) were presented at 12 months (primary endpoint), 24 months, and have now persisted through 36 months following subretinal administration of OpRegen cell therapy
  • Mean change in BCVA among treated eyes for Cohort 4 patients (n=10) completing 3-year follow up was +6.2 letters (compared to +5.5 letters at 24 months) (Early Treatment Diabetic Retinopathy Study (ETDRS) assessment)
  • Effects were greater on average in the five (5) Cohort 4 patients with extensive OpRegen cell therapy coverage of atrophic areas at the time of surgical delivery
    • In these patients’ treated eyes, the mean change in BCVA was +9.0 ETDRS letters for those completing 3-year follow-up (compared to +7.4 ETDRS letters at 24 months) (n=5)
  • Sustained evidence of retinal structural improvement by a quantitative Optical Coherence Tomography (OCT) analysis through 36 months was observed in treated eyes of Cohort 4 patients following a single subretinal administration of OpRegen cell therapy
  • At month 36, sustained evidence of retinal structure improvements in external limiting membrane (ELM) and RPE complex (RPE-C) layers on OCT was observed in the subgroup of five (5) patients in Cohort 4 with extensive OpRegen cell therapy bleb coverage of atrophic areas at the time of surgical delivery
    • Mean improvement of RPE-C area compared with baseline was maintained in treated eyes from 24 months (+2.6 mm2; n=4) to 36 months (+1.9 mm2; n=5)
      • In comparison, mean change in RPE-C area decreased in untreated fellow eyes from 24 months (-2.8 mm2; n=4) to 36 months (-3.8 mm2; n=5)
    • Mean change in ELM area was maintained in treated eyes from 24 months (+0.8 mm2; n=4) to 36 months (+0.3 mm2; n=5)
      • In comparison, mean change in ELM area decreased in untreated fellow eyes from 24 months (-1.9 mm2; n=4) to 36 months (-3.4 mm2; n=5)
  • Imaging evidence of structural benefit and greater gains in visual acuity were seen in study eyes in Cohort 4 patients at month 12, continuing through month 36
  • Differences as compared with fellow eyes increased over time, suggesting possible modification of the course of disease
  • Quantitative OCT improvements in RPE-C and ELM area and the gains in BCVA were more prominent in patients with extensive compared with limited OpRegen bleb coverage of GA
  • Quantitative analysis of OCT imaging revealed areas with partial restoration of outer retinal structure including re-appearance of an RPE layer as well as features associated with recovery of photoreceptors
  • These data suggest that OpRegen cell therapy may counteract RPE cell dysfunction and loss in GA by providing support to remaining retinal cells, and these effects appear durable through at least 36 months after a single administration
  • The Phase 2a “GAlette study” evaluating the success of subretinal delivery of OpRegen cell therapy to target areas of GA is currently enrolling (NCT05626114)
    • In addition to evaluating other surgical parameters, this study will test proprietary surgical devices in development for subretinal delivery of OpRegen cell therapy that have potential advantages over currently available devices and procedures