OpRegen®
for Dry AMD
OpRegen® (RG6501) is an allogeneic retinal pigmented epithelial (RPE) cell replacement therapy currently in Phase 2a development under a worldwide collaboration with Roche and Genentech for the treatment of geographic atrophy (GA) secondary to dry-AMD.
Our approach is to replace the layer of damaged RPE cells with new, healthy, and functional RPE cells manufactured from a well-characterized, allogeneic cell line, transplanted to the subretinal space, and extensively cover the area of geographic atrophy.
The goal of the OpRegen therapeutic approach is to slow or halt disease progression and to preserve and/or restore visual function in patients affected by dry-AMD. OpRegen has been granted Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA, which includes an expedited regulatory path with the ability for increased interfacing with the FDA during the clinical development process.
Image adapted from scienceofamd.org
The GAlette (Phase 2a) Study
OpRegen is currently being evaluated in a Phase 2a study intended to optimize subretinal surgical delivery and evaluate safety and activity in up to 60 patients. The primary objectives of the study are to evaluate (i) the proportion of patients with subretinal surgical delivery of OpRegen to target regions under the retina, and (ii) to evaluate the safety of subretinal surgical delivery of OpRegen as measured by the incidence and severity of procedure-related adverse events at 3 months following surgery. A key secondary objective is to evaluate the proportion of patients with qualitative improvement in retinal structure, as determined by Optical Coherence Tomography (SD-OCT) imaging, within 3 months following surgery.
Clinical Data
In June 2025, 36-month visual acuity results from patients enrolled in a Phase 1/2a clinical study of OpRegen in patients with GA secondary to AMD, were presented at the Clinical Trials at the Summit 2025.
- Mean vision gains of +9 letters among patients with extensive coverage of OpRegen cell therapy to the GA lesion site
- Evidence of retinal structural improvement persisted out to 3 years
- Anatomical and functional improvements occur following a single administration of OpRegen cell therapy
Clinical Trials at the Summit 2025 Highlights
- Improvement in visual acuity in Cohort 4 patients (less advanced GA than in other cohorts) was present at 12 months (primary endpoint), 24 months, and has now persisted through 36 months
- Gains in Best Corrected Visual Acuity (BCVA) in patients in Cohort 4 (less advanced GA) measured at month 12 remain evident through month 36 following subretinal administration of OpRegen cell therapy
- Mean change in BCVA among treated eyes for 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)
- Improvement in BCVA and outer retinal structure in patients with extensive OpRegen bleb coverage of their GA area was greater than in patients with limited coverage and persisted through month 36
- Effects were greater on average in the five (5) 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 (less advanced GA than in other cohorts) 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 drusen complex (RPEDC) layers on OCT was observed in the subgroup of five patients in Cohort 4 with extensive OpRegen cell therapy bleb coverage of atrophic areas at the time of surgical delivery
- Mean improvement of RPEDC 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 RPEDC 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)
- Mean improvement of RPEDC 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)
- These data suggest that OpRegen cell therapy may counteract RPE cell dysfunction and loss in GA by providing support to the remaining retinal cells within atrophic areas, 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 Clinical Trials at the Summit 2025 presentation is available on the Events and Presentations section of Lineage’s website.
BCVA gains in study eyes are sustained
in Cohort 4 (Less advanced GA) patients 36 Months Post-Treatment and BCVA Gains are Greater Among Eyes with Extensive Coverage of GA by the Surgical Bleb
Presented at the Clinical Trials at the Summit 2025
Sustained Evidence of Retinal Structural Support
in Cohort 4 (Less advanced GA) patients 36 Months Post-Treatment by Quantitative OCT Analysis
Sustained Evidence of Retinal Structural Support is Most Evident Among Eyes with Extensive Coverage of GA by the Surgical Bleb
Presented at the Clinical Trials at the Summit 2025
GAllete Study Design
A Phase 2a Multicenter, Open-Label, Single-Arm Study to Optimize the Subretinal Delivery of Opregen Cell Therapy In GA (NCT05626114)
Presented at the Clinical Trials at the Summit 2025
Advanced Subretinal Delivery Devices
Under Development for Evaluation with OpRegen Cell Therapy
Presented at the Clinical Trials at the Summit 2025