Ocular Diseases
Ceregene is developing novel treatments for several serious ocular diseases. Retinitis pigmentosa (RP) is one such disease. RP is caused by many different genetic mutations, as well as possible environmental factors, which lead to 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. Tragically, all patients eventually become blind, for there is no treatment or cure. This disease is often recognized in children, adolescents and young adults, with progression of the disease continuing throughout the individual’s life. Clearly, there is a desperate need for pioneering therapy for this disease. Following initiation of a clinical trial for RP, Ceregene plans to explore treatments for other blinding ocular diseases, such as wet and dry age-related macular degeneration (AMD) and glaucoma.
Ceregene's Treatment Strategy for Ocular Diseases
Ceregene is using its novel gene delivery technology to deliver the gene for neurotrophic factor 4 (NT4) to the retina. This product, CERE-140, is still in nonclinical (not yet testing in humans) stages of development, but initial animal studies injecting CERE-140 into the large chamber of the eye, have salvaged and improved the function of photoreceptors in animal models of RP. CERE-140 is thus intended to enhance the function and prevent loss of photoreceptor cells in the retina, restoring and preserving sight in patients with RP. A major advantage of our approach is that it should be feasible to treat all cases of RP, independent of which different genetic mutation is responsible for the disease. Moreover, a single dosing session with CERE-140 may be sufficient to maintain and possibly restore sight, indefinitely.
This same approach should be useful in treating both the dry and wet forms of age-related macular degeneration (AMD), since loss of these same photoreceptors is a major cause of blindness in AMD. While current therapies for the wet form of macular degeneration are able to treat the abnormal blood vessels that form and obscure vision due to their leakage, no current therapy is able to delay or halt the degeneration of the photoreceptors, in either the wet or dry form of AMD. It is the loss of photoreceptors that is the more direct cause of blindness in AMD and thus an attractive target for innovative neurotrophic factor therapy.
Similarly, no treatment is yet available for the cell loss and the degeneration of the optic tract that occurs in glaucoma. Because CERE-140 also has the potential to restore function and protect the cells in the eye that comprise the optic nerve, we are also testing CERE-140 in nonclinical (animal) models of glaucoma.
In sum, our approach to treating ocular diseases using NT4, in conjunction with our proprietary gene delivery technology, may provide 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, amyotrophic lateral sclerosis (ALS), Huntington’s disease and several other serious neurodegenerative diseases.