Lineage Cell Therapeutics, Inc. announced that 36-month results from patients enrolled in a Phase 1/2a clinical study (ClinicalTrials.gov Identifier: NCT02286089) of RG6501 (OpRegen) in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD), were presented at the Foundation Fighting Blindness? Retinal Therapeutics Innovation Summit 2026. Gains in best corrected visual acuity (BCVA) of +9 letters at 36 months among patients with extensive coverage of OpRegen cell therapy to the geographic atrophy (GA) lesion site.
OCT imaging indicated partial restoration of outer retinal structure, re-appearance of an RPE layer, and features associated with recovery of photoreceptors. Anatomical and functional improvements occur following a single administration of OpRegen cell therapy. RG6501 (OpRegen) is a suspension of human allogeneic retinal pigment epithelial (RPE) cells currently in development for the treatment of GA secondary to AMD.
Subretinal delivery of OpRegen cell therapy has the potential to counteract RPE cell loss in areas of GA lesions by supporting retinal cell health and improving retinal structure and function. It is being developed under an exclusive worldwide collaboration between Lineage, Roche, and Genentech, a member of the Roche Group, and is currently being evaluated in a Phase 2a clinical study, GAlette, in patients with GA secondary to AMD (ClinicalTrials.gov Identifier: NCT05626114). 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.
The Phase 1/2a study is an open-label, single-arm, multi-center, dose-escalation trial evaluating a single administration of OpRegen cell therapy delivered subretinally in patients with bilateral GA secondary to AMD. Twenty-four patients were enrolled into 4 cohorts. The first 3 cohorts enrolled only legally blind patients with a best corrected visual acuity (BCVA) of 20/200 or worse.
The fourth cohort enrolled 12 patients with impaired vision (BCVA from 20/65 to 20/250 with smaller mean areas of GA). Cohort 4 also included patients treated with a new ?thaw-and-inject? formulation of OpRegen cell therapy, which can be shipped directly to sites and used immediately upon thawing, removing the complications and logistics of having to use a dose preparation facility.
The primary objective of the study was to evaluate the safety and tolerability of OpRegen cell therapy as assessed by the incidence and frequency of treatment-emergent adverse events. Secondary objectives include evaluating the preliminary activity of OpRegen cell therapy treatment by assessing the changes in ophthalmological parameters measured by various methods of primary clinical relevance.
