Lineage Cell Therapeutics (LCTX) presented the 36‑month results from its Phase 1/2a study of RG6501 (OpRegen) at the Foundation Fighting Blindness Retinal Therapeutics Innovation Summit in Carlsbad, California, on May 1, 2026.
The data show that patients who received OpRegen experienced durable improvements in best‑corrected visual acuity (BCVA) over three years, with a mean gain of +9 ETDRS letters in treated eyes that maintained or exceeded baseline BCVA at 12, 24, and 36 months. The previously reported 20‑letter gain was an overstatement; the most consistently cited figure is the +9‑letter improvement for patients with extensive coverage of the atrophic area.
The study, registered under ClinicalTrials.gov identifier NCT02286089, enrolled 24 participants across four cohorts and evaluated a single subretinal injection of allogeneic retinal pigment epithelial (RPE) cells. Cohort 4, which received a thaw‑and‑inject formulation, provided the most robust data on long‑term safety and efficacy.
OpRegen delivers a single dose of RPE cells designed to replace lost or dysfunctional cells in the retina, aiming to restore structural integrity and visual function in geographic atrophy (GA). The therapy’s regenerative mechanism distinguishes it from the complement‑inhibitor drugs currently approved for GA, which primarily slow disease progression without restoring vision.
The 36‑month milestone is significant because it represents the longest‑term evidence of OpRegen’s safety and efficacy to date, reinforcing Lineage’s development plan and the partnership with Roche/Genentech. Roche has committed substantial upfront and milestone payments—potentially up to $670 million—underscoring confidence in the program’s commercial potential.
Lineage CEO Brian M. Culley said, "Long term clinical outcomes following a single administration of OpRegen cell therapy is challenging the long‑held view that GA causes irreversible damage. A key finding from the Lineage‑run phase 1/2a trial was the importance of extensive placement of cells across the area of atrophy. Among patients who received fulsome coverage by OpRegen cell therapy, anatomical and functional benefits from a single administration have lasted for at least three years." He added, "Over this extended period, improved visual function is not a natural occurrence among patients suffering from GA, making these changes quite promising. Importantly, OpRegen‑treated eyes have exhibited mean BCVA scores above baseline at each of the 12‑, 24‑, and 36‑month timepoints, demonstrating consistency as well as durability."
The data position OpRegen as a potentially transformative therapy in a market dominated by complement inhibitors such as pegcetacoplan (Syfovre) and avacincaptad pegol (Izervay), which have not shown visual restoration. A single‑administration, cell‑based approach could offer a unique therapeutic advantage and address an unmet need for vision improvement in GA patients.
Overall, the presentation of 36‑month data at the summit marks a pivotal moment for Lineage and its Roche partnership, providing the most compelling long‑term evidence to date that a regenerative cell therapy can deliver sustained visual gains in geographic atrophy.
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