Neurotrophic Factors

Neurotrophic factors provide the opportunity to enhance the vitality and function of dying neurons in the brain and eyes while also enabling the neurons to withstand further degeneration. They are naturally occurring proteins known to have powerful beneficial effects on neurons, though until now, the lack of a means to deliver them to the degenerating neurons has hampered their development as therapies for human diseases. Ceregene’s gene delivery technology is intended to provide targeted, long-term exposure of dying neurons to specific neurotrophic factors known to help those neurons. Thus, Ceregene’s products offer the opportunity to both substantially improve the serious symptoms, but also delay the progression of Parkinson’s disease, Alzheimer’s disease, ALS (Lou Gehrig’s disease) and several ocular diseases that cause blindness.

Therapeutic Targets

Improving the function while protecting dying neurons remains a very challenging medical problem. The causes of neurodegeneration range widely and include injury, hereditary defects, exposure to toxins, neurodegenerative disorders, and age-related neurodegeneration. The effects are often devastating and include paralysis, loss of motor skills, blindness and serious loss of mental function.

Treatment

Advances in biology have uncovered naturally occurring biological proteins (called neurotrophic factors) that are responsible for maintaining the viability of cells in the brain and the eyes. All these neurotrophic factors are crucial to the survival of the neurons early in the brain’s development. Without them, the neurons simply will not make proper connections and cannot survive. In the adult brain, they take on more of a maintenance function. In situations where these cells are degenerating, such as occurs with disease, these potent neurotrophic factors are able to restore lost function, regenerate damaged cells, and protect against further damage and cell death.

Collectively, neurotrophic factors have become a resource for possible treatments for injuries or pathological conditions that affect the nervous and ocular systems. Because the neurotrophic factors are large proteins, they cannot be taken orally, are not stable in systemic circulation and cannot access the brain or eye where the degenerating cells reside. For this reason, lack of an effective means of delivering the growth factors to the targeted sites of neurodegeneration has kept them from being developed as effective therapeutics for human disease.

Ceregene has developed gene delivery technology as an alternative means to administer growth factors, with the added benefit of prolonged effects from only a single treatment. By delivering the neurotrophic gene to a highly specific targeted area where neurodegeneration is occurring, a constant source of the neurotrophic factor is provided precisely to the tissues most damaged by the disease.

Ceregene Core Technology

Ceregene has identified several neurotrophic factors that can restore the vitality of neurons in different brain and eye diseases, as well as help protect these neurons from further degeneration. Ceregene has also developed proprietary gene delivery technology that allows delivery of the appropriate neurotrophic factor gene to those cells that require it for proper function and survival, thus providing novel treatments for diseases as varied as Parkinson’s disease, Alzheimer’s disease, ALS (Lou Gehrig’s disease) and several ocular diseases that cause blindness.