Gene Delivery Technology

Gene Delivery Technology

Ceregene has developed the technology to permit the genes for different neurotrophic factors to be transferred to the neurons that are degenerating in various diseases. This enables these neurons to produce the neurotrophic factors that will restore their vitality and function while also helping them overcome further degeneration. Moreover, this approach provides the means of selectively targeting only those regions of the brain or eye that require the neurotrophic factor. Additionally, a single treatment is all that is required to provide prolonged, and perhaps permanent, therapeutic benefit to large numbers of patients with neurodegenerative and ocular degenerative diseases. Thus, Ceregene’s gene delivery technology solves an age-old problem of how to effectively deliver these potent neurotrophic factors that hold the key to both significantly reducing the serious symptoms, but also protecting against further degeneration in several terrible diseases, such as Parkinson’s disease, Alzheimer’s disease, ALS (Lou Gehrig’s disease) and several ocular diseases that cause blindness.

How Ceregene’s Gene Delivery Technology Works

Gene transfer offers an effective means of bathing diseased and dying neurons with neurotrophic factors in a targeted and prolonged fashion. Ceregene has identified several neurotrophic factors to treat damaged nerve cells in several different diseases. For each disease, the appropriate gene encoding the neurotrophic factor known to provide nourishment for the key cells that are dying is delivered to those cells in a single dosing procedure. This is accomplished through a vector we created that delivers the therapeutic gene to the target cells. Once the customized vector delivers the therapeutic gene into the cell, these cells are able to continuously produce the neurotrophic factor. Elevated levels of the neurotrophic factor enhance the vitality and function of the degenerating neurons while also helping them withstand further damage from the disease.

Ceregene uses a harmless version of a virus that infects humans, adeno-associated virus (AAV) type 2 as the vector to carry the neurotrophic factor genes to the target tissues. Interestingly, most humans have been exposed to the AAV type 2 virus naturally, and this virus is not known to be associated with any human disease, even in its naturally-occurring virulent form. Nonetheless, Ceregene has modified AAV to assure it cannot replicate, or reproduce itself, thus assuring that the genes we deliver remain in the targeted tissue only and thus the neurotrophic factors they produce are elevated only in those sites where they are needed to treat the disease.

Ceregene’s AAV-based gene delivery technology provides a wide platform to develop treatments for many serious but different diseases, including Alzheimer’s Disease, Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig’s disease), Parkinson’s Disease, and Ocular Diseases). Some of these have reached investigative new drug (IND) status and have entered clinical trials.