Ocular Diseases

Ceregene is developing novel treatments for several ocular diseases in order to restore lost vision and prevent eventual blindness. Retinitis pigmentosa (RP) is one such serious disease. It is inherited due to many different genetic mutations, causing the degeneration of photoreceptor cells (rods and cones) in the retina. Early symptoms of this disease include color and night blindness, tunnel vision, blurred vision, or shadows; eventually patients become blind. This disease is most often recognized in children, adolescents and young adults, with progression of the disease continuing throughout the individual’s life. There are no current treatments for this disease.

Ceregene Treatments for Degenerative Eye Disease

Ceregene is using its novel gene delivery technology to delivery the genes for selected neurotrophic factors directly to the retina. While this program is still in preclinical (not yet testing in humans) stages of development, initial animal studies using targeted delivery of genes for these neurotrophic factors to retinal tissue have yielded positive results. These treatments are designed to enhance the function and prevent loss of photoreceptive cells in the retina, thereby restoring and preserving sight in patients with RP. A major advantage of Ceregene’s approach is that this is feasible independent of the possibility that many different genetic mutations might be responsible for the disease in any particular patient. Moreover, a single dosing session with Ceregene’s product is all that is needed to maintain and possibly restore sight, indefinitely.

It is believed this same approach will be useful in treating wet and dry forms of age-related macular degeneration and blindness caused by ocular hypertension in patients with glaucoma. While current therapies for the wet form of macular degeneration are able to retard the rate of abnormal vessel formation and their leakage (which contributes to the loss of vision in these patients), no current therapy is able to truly delay or halt the degeneration of the photoreceptors, which is the direct cause of blindness. Similarly, no treatment is yet available for the cell loss in the dry form of macular degeneration or for the degeneration of the optic tract that occurs in glaucoma.

Thus, Ceregene’s approach to treating ocular diseases using neurotrophic factors via its proprietary gene delivery technology provides the best means of restoring vision and preventing blindness in a range of ocular diseases. This same technological platform provides an opportunity to restore function and prevent disease progression in Parkinson’s disease, Alzheimer’s disease, ALS (Lou Gehrig’s disease) and several other serious neurodegenerative diseases.