Lineage Cell Therapeutics announced today that 36-month results from the Phase 1/2a clinical trial of RG6501 (OpRegen) will be presented at the upcoming Clinical Trials at the Summit (CTS) 2025 conference. The data presentation will showcase long-term outcomes in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD), a condition that represents a significant unmet medical need as a leading cause of adult blindness worldwide.
Dr. Christopher D. Riemann, Vitreoretinal Surgeon and Fellowship Director at Cincinnati Eye Institute and University of Cincinnati School of Medicine, will present the findings on behalf of Roche and Genentech at the conference scheduled for June 20-21, 2025, in Las Vegas.
Novel Cell Therapy Approach for Geographic Atrophy
OpRegen represents an innovative approach to treating GA, a condition that affects at least 5 million people globally. The therapy consists of a suspension of allogeneic retinal pigment epithelial (RPE) cells delivered subretinally to potentially counteract RPE cell loss, which is characteristic of GA. By supporting retinal cell health and improving retinal structure and function, OpRegen aims to address the underlying pathology of the disease rather than merely managing symptoms.
"Geographic atrophy represents a significant cause of vision loss for which we currently have limited treatment options," said Dr. Riemann in a statement regarding the upcoming presentation. "The 36-month data from this trial provides valuable insights into the long-term safety and potential efficacy of cell replacement therapy in this patient population."
Phase 1/2a Study Design and Patient Population
The open-label, single-arm, multi-center, dose-escalation trial evaluated a single administration of OpRegen delivered subretinally in patients with bilateral GA secondary to AMD. Twenty-four patients were enrolled across four cohorts with varying degrees of vision impairment:
- Cohorts 1-3: Included only legally blind patients with best corrected visual acuity (BCVA) of 20/200 or worse
- Cohort 4: Enrolled 12 patients with less severe vision impairment (BCVA from 20/65 to 20/250) and smaller mean areas of GA
Notably, Cohort 4 utilized a "thaw-and-inject" formulation of OpRegen that simplifies logistics by allowing direct shipment to clinical sites without requiring specialized dose preparation facilities.
The primary objective focused on safety and tolerability, with secondary objectives evaluating preliminary activity through changes in ophthalmological parameters. While complete results will be presented at the conference, the fact that 36-month data is being shared suggests the treatment has maintained a safety profile that supports continued development.
Collaborative Development Efforts
OpRegen is being developed under an exclusive worldwide collaboration between Lineage Cell Therapeutics and Roche/Genentech. The therapy is currently advancing in a separate Phase 2a clinical study (NCT05626114) in patients with GA secondary to AMD.
"The presentation of this long-term data represents an important milestone in our development program for OpRegen," noted Lineage Cell Therapeutics in their announcement. "Geographic atrophy remains an area of significant unmet need, and we are committed to advancing potential new treatment options for these patients."
Geographic Atrophy: A Significant Unmet Need
GA is an advanced form of AMD characterized by severe loss of visual function. It typically affects both eyes and can occur simultaneously with neovascular AMD, the "wet" form of the disease. Importantly, patients treated for neovascular AMD may still develop GA, highlighting the need for specific therapies targeting this condition.
The disease burden is substantial, affecting millions globally with limited treatment options currently available. Cell-based approaches like OpRegen represent a novel therapeutic strategy that could potentially address the underlying cellular degeneration rather than just slowing disease progression.
About Lineage Cell Therapeutics
Lineage Cell Therapeutics is developing the OpRegen program as part of its broader portfolio of "off-the-shelf" cell therapies derived from pluripotent cell lines. The company's platform focuses on creating specialized human cells with functions similar to those found naturally in the body, which are then transplanted to replace or support cells damaged by disease or injury.
Beyond OpRegen, Lineage's neuroscience-focused pipeline includes OPC1 for spinal cord injuries, ReSonance™ for auditory neuropathy, PNC1 for vision loss due to photoreceptor dysfunction, and RND1, a novel hypoimmune induced pluripotent stem cell line.
The upcoming presentation at CTS 2025 will provide the scientific community with important insights into the potential long-term benefits of this innovative cell therapy approach for patients suffering from geographic atrophy, a condition with significant impact on quality of life and independence for affected individuals.
