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Inherited retinal diseases (IRDs) are a group of diseases that can lead to severe vision loss or even blindness. To date, IRDs are known to be associated to over 270 genes. IRDs can affect individuals of all ages, can progress at different rates, and are rare. IRDs damage the rod and/or cone photoreceptor cells in the retina. Many IRDs are degenerative, which means that the symptoms of the disease will get worse over time.
Retinitis pigmentosa (RP) is a group of eye disorders caused by mutations in over 90 genes that affect the retina. In individuals with RP, the light-sensing cells of the retina gradually die off, leading to vision loss. The severity and progression of the disease can vary based on the specific gene affected. RP may first appear in childhood (early onset RP) or adulthood, with the initial symptom often being night blindness. As the condition progresses, blind spots develop in peripheral vision, eventually affecting central vision and impacting activities like reading, driving, and recognizing faces.
Vision changes in RP patients
Photoreceptor cell death is a major contributor to visual loss in retinal degenerative disorders, such as Retinitis Pigmentosa, Stargardt disease, and dry AMD.
Optogenetic gene therapy offers promising potential for individuals suffering from inherited retinal degenerative diseases. Optogenetics utilizes gene therapy to deliver optogenetic genes to retinal cells. These optogenetic genes enable retinal cells to produce proteins that respond to light. When light enters the eye, it activates the optogenetic proteins, which produce visual signals that are sent to the brain. As a result, vision in patients with retinal degenerative diseases can be reconstructed.
ZM Therapeutics' optogenetic gene therapy utilizes adeno-associated virus (AAV) to deliver a gene encoding an ambient light-activated protein into retinal cells. The therapy is designed for administration through a safe and convenient intravitreal injection, with the potential to address all forms of retinal degeneration. ZM Therapeutics' optogenetic products represent a truly next-generation therapy, enabling retinal cells to detect light, offering patients with photoreceptor degenerative diseases the possibility to regain their vision.
Our lead optogenetic product is currently in clinical development for retinitis pigmentosa (RP). A dose-escalation, randomized controlled study is underway. Patients will receive a single intravitreal injection, followed by one year of observational follow-up.
