OpRegen® 3-Year Data in Geographic Atrophy Patients Showcased at Foundation Fighting Blindness Retinal Innovation Summit 2026
Lineage Cell Therapeutics (NYSE American and TASE: LCTX), a clinical-stage biotechnology company focused on developing off-the-shelf allogeneic cell therapies for serious medical conditions, has reported encouraging 36-month data from its Phase 1/2a clinical study evaluating RG6501 (OpRegen) in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The findings were presented at the Retinal Therapeutics Innovation Summit 2026, a key forum for advancements in retinal disease research and innovation.
The presentation, delivered by Eyal Banin, M.D., Ph.D., of the Hadassah-Hebrew University Medical Center, showcased long-term clinical outcomes from patients treated with OpRegen, a retinal pigment epithelium (RPE) cell therapy. The study was conducted in collaboration with Roche and Genentech, highlighting the ongoing partnership aimed at advancing regenerative treatments for retinal diseases.
Geographic atrophy, an advanced form of AMD, is a progressive and irreversible condition characterized by the degeneration of retinal cells, particularly the retinal pigment epithelium. This leads to gradual and permanent vision loss, with limited treatment options available. Current therapies for GA primarily focus on slowing disease progression, but they have not demonstrated meaningful improvements in visual function. Against this backdrop, the potential for a therapy that can restore retinal structure and improve vision represents a significant breakthrough.
OpRegen is a suspension of human allogeneic RPE cells designed to replace damaged or lost RPE cells in the retina. Delivered via a subretinal surgical procedure, the therapy aims to support the health and function of surrounding retinal cells, including photoreceptors, which are essential for vision. By addressing the underlying cellular damage, OpRegen has the potential to alter the course of the disease rather than merely slowing its progression.
According to Brian Culley, Chief Executive Officer of Lineage, the latest data reinforce the company’s confidence in the therapeutic potential of OpRegen. He emphasized that the durability of the observed benefits over three years is particularly noteworthy, as it challenges the long-standing assumption that geographic atrophy is an irreversible condition. Culley also highlighted that the therapy’s ability to deliver meaningful outcomes following a single administration could represent a paradigm shift in the treatment of retinal degenerative diseases.
The 36-month results presented at the summit focused on patients in Cohort 4 of the study, a group characterized by less advanced disease at baseline compared to earlier cohorts. Among these patients, sustained improvements in best corrected visual acuity (BCVA) were observed over the three-year follow-up period. On average, treated eyes demonstrated a gain of +6.2 letters on the Early Treatment Diabetic Retinopathy Study (ETDRS) scale, compared to a +5.5-letter improvement at 24 months. These gains are significant in the context of GA, where vision typically declines over time.
Even more pronounced improvements were observed in a subgroup of patients who received extensive coverage of the atrophic area with OpRegen cells during the surgical procedure. In this group, the mean improvement in BCVA reached +9.0 ETDRS letters at 36 months, compared to +7.4 letters at 24 months. These findings suggest that the extent of cell coverage may play a critical role in maximizing therapeutic benefit, pointing to the importance of optimizing delivery techniques.
In addition to functional improvements, the study provided compelling evidence of structural changes within the retina. Quantitative analyses using optical coherence tomography (OCT) revealed sustained improvements in key retinal layers, including the external limiting membrane (ELM) and the RPE complex (RPE-C). These structural enhancements were observed through 36 months following a single administration of the therapy, indicating long-term stability and potential regeneration of retinal tissue.
Among patients with extensive OpRegen coverage, the mean improvement in RPE-C area was maintained from +2.6 mm² at 24 months to +1.9 mm² at 36 months. In contrast, untreated fellow eyes showed continued deterioration, with mean changes declining from -2.8 mm² to -3.8 mm² over the same period. Similar trends were observed in the ELM area, where treated eyes maintained improvements while untreated eyes experienced progressive loss. These comparative findings underscore the potential of OpRegen to not only halt but potentially reverse aspects of retinal degeneration.
Imaging data further revealed partial restoration of outer retinal structures, including the reappearance of an RPE layer and features associated with photoreceptor recovery. Such observations are particularly significant, as they suggest that the therapy may be capable of re-establishing critical components of the visual pathway. The persistence of these effects over three years supports the durability of the treatment and its potential to provide long-lasting benefits.
Another important observation from the study was the increasing divergence between treated and untreated eyes over time. This widening gap suggests that OpRegen may influence the natural progression of the disease, offering evidence of disease modification rather than temporary improvement. The consistency of both structural and functional gains strengthens the case for further clinical development.
The ongoing Phase 2a GAlette study is designed to build on these findings by optimizing the surgical delivery of OpRegen. This study is currently enrolling patients and aims to evaluate various procedural parameters, including the use of proprietary surgical devices developed specifically for subretinal delivery. These devices are intended to improve precision, enhance cell coverage, and potentially increase the overall effectiveness of the therapy.
The collaboration between Lineage, Roche, and Genentech continues to play a central role in advancing OpRegen through clinical development. By combining expertise in cell therapy, ophthalmology, and global drug development, the partnership is well positioned to address the complex challenges associated with treating retinal degenerative diseases.
The presentation at the Retinal Therapeutics Innovation Summit 2026 represents an important milestone in the development of OpRegen. The accumulation of long-term data provides valuable insights into the therapy’s safety, efficacy, and durability, while also informing future study design and regulatory strategy. As additional data emerge, they will further clarify the potential role of OpRegen in the treatment landscape for geographic atrophy.
In summary, the 36-month results from the Phase 1/2a study of OpRegen highlight the promise of RPE cell therapy as a transformative approach to treating GA secondary to AMD. The observed improvements in visual acuity, coupled with sustained structural restoration of retinal tissue, suggest that this therapy may offer benefits beyond those achievable with current treatments. As research continues, OpRegen has the potential to redefine expectations for patients with geographic atrophy, offering hope for improved vision and quality of life in a condition that has long been considered untreatable.
About the Retinal Innovation Summit 2026
The annual Retinal Therapeutics Innovation Summit 2026 is jointly organized by the Foundation Fighting Blindness and the Oregon Health & Science University Casey Eye Institute. Members of the medical and research communities will come together with representatives from the biotech and pharma industries to discuss rapidly emerging ocular therapies and strategize how to remove barriers toward clinical utility for the most advanced retinal disease therapies.
The Retinal Therapeutics Innovation Summit features presentations by leading experts on potential therapeutic approaches to treat retinal diseases and how best to deliver them to patients. In this congenial setting, summit attendees discuss progress being made in the field and how to use initial clinical successes to move toward larger scale trials. For more information, visit: https://www.fightingblindness.org/events/innovation-summit-2026-4761.
About the OpRegen Phase 1/2a Study
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.
About Geographic Atrophy
GA is an advanced form of AMD characterized by severe loss of visual function. GA is a leading cause of adult blindness in the developed world, affecting at least 5 million people globally. There are two forms of advanced AMD: neovascular AMD and GA. GA and neovascular AMD can occur simultaneously in the same eye, and patients treated for neovascular AMD may still go on to develop GA. GA typically affects both eyes.
About Lineage Cell Therapeutics, Inc.
Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel allogeneic, or “off the shelf”, cell therapies for serious medical conditions. Lineage’s programs are based on its proprietary cell-based technology platform, AlloSCOPE™ (Allogeneic, Scalable, Consistent, Off-the-shelf, Pluripotent Cell Engineering), and associated development and manufacturing capabilities. From this proprietary AlloSCOPE platform, Lineage develops, manufactures, and tests specialized human cells with anatomical and physiological functions similar or substantially identical to cells found naturally in the human body. These cells are created by applying directed differentiation protocols to established, well-characterized, and self-renewing pluripotent cell lines.
These protocols generate cells with characteristics associated with specific and desired developmental lineages, and in some instances may be designed to have additional beneficial properties. Cells derived from such lineages are transplanted into patients in an effort to replace or support cells that are absent or dysfunctional due to degenerative disease, aging, or traumatic injury, and to restore or augment the patient’s functional activity.
Lineage’s pipeline currently includes: (i) OpRegen® cell therapy, a retinal pigment epithelial cell therapy in Phase 2a development under a worldwide collaboration with Roche and Genentech, a member of the Roche Group, for the treatment of geographic atrophy secondary to age-related macular degeneration;
(ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of spinal cord injuries;
(iii) ReSonance™ (ANP1), an auditory neuronal progenitor cell therapy in preclinical development under a collaboration with William Demant Invest A/S for the potential treatment of auditory neuropathy;
(iv) PNC1, a photoreceptor neural cell therapy research initiative being evaluated for development for the potential treatment of vision loss due to photoreceptor dysfunction or damage;
(v) RND1, a novel hypoimmune induced pluripotent stem cell line being evaluated for development under a gene editing partnership;
(vi) ILT1, a cell therapy research initiative focused on the issue of large-scale production of undifferentiated pluripotent cells, which if successful could be evaluated for the production of islet cells to support a potential treatment of Type 1 Diabetes; and (vii) COR1, a corneal endothelial disease cell therapy in preclinical development for the potential treatment of corneal endothelial disease.
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