Bioengineered optogenetic medicines to preserve and restore vision for degenerative retinal diseases.
We are advancing our pipeline of optogenetic medicines to address a range of degenerative retinal diseases, with programs in both preclinical and clinical stages of development.
Degenerative retinal diseases encompass a variety of conditions that cause progressive damage to photoreceptors, the cells lining the retina that respond to light and send visual signals to the brain. Each disease has a different pattern of damage, but all result in progressive vision loss that can lead to blindness.
The physical and emotional toll of vision loss is immense. It impacts independence, mobility, and daily tasks like reading and recognizing faces. People often face increased risk of injury, reduced job opportunities, and educational challenges. Emotionally, vision loss can lead to feelings of helplessness, fear, anxiety, and social isolation. It’s also linked to higher rates of physical inactivity and health issues like diabetes and heart disease.
Hover over each image to view a simulated representation of vision loss associated with the condition.
Simulated vision of peripheral vision loss: clear central focus with darkened, blurred edges representing lost peripheral sight
Retinitis pigmentosa (RP) can be caused by a mutation in one of dozens of genes and affects the entire retina.
The condition is typically diagnosed in the first decades of life, and symptoms include night blindness, reduced visual fields, loss of visual acuity, and, eventually, complete or near-complete blindness. Worldwide, RP is estimated to impact more than half a million people, and no effective treatments are available.
Additional Resource:
American Academy of Ophthalmology
Simulated vision of peripheral vision loss: clear central focus with darkened, blurred edges representing lost peripheral sight.
Choroideremia
Choroideremia is an inherited, X-linked form of retinal degeneration caused by mutations in the CHM gene.
Symptoms include night blindness, loss of visual acuity, and progressive loss of peripheral vision. Choroideremia affects as many as 1 in 50,000 males worldwide and has no effective treatments.
Additional Resource:
Fighting Blindness
Simulation of central vision loss: central visual field appears blurred or obscured, with peripheral vision remaining intact and relatively unaffected.
Stargardt’s Disease
Stargardt’s Disease is an inherited retinal degeneration that can manifest in childhood or adolescence.
People lose central vision and visual acuity due to degeneration of the macula, the area at the center of the retina. Stargardt’s disease is estimated to impact about 1 in 17,000 people worldwide, and there are no effective treatments.
Additional Resource:
American Academy of Ophthalmology
Simulation of central vision loss: central visual field appears blurred or obscured, with peripheral vision remaining intact and relatively unaffected.
Geographic Atrophy
Geographic atrophy (GA) is an advanced form of age-related macular degeneration (AMD) whose causes are not fully understood.
GA results in the loss of central vision and visual acuity due to degeneration of the macula. It impacts about 20 percent of people with AMD and more than 8 million people worldwide.
Additional Resource:
Prevent Blindness
RTx-015 for Retinitis Pigmentosa or Choroideremia
Phase 1
This Phase 1 trial, ENVISION, will evaluate the safety and preliminary efficacy of a single intravitreal injection into one eye of RTx-015, our investigational optogenetic therapy, in patients with RP or Choroideremia.
ENVISION is an open-label, non-randomized, and multicenter trial that will enroll approximately 15 patients. For more information about ENVISION, please visit https://clinicaltrials.gov/study/NCT06460844.
Learn how RTX-015 repurposes retinal ganglion cells to restore vision.
Learn how RTX-021 repurposes retinal ON-bipolar cells to restore vision.
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