Gene Delivery Technology
Ceregene is developing several pioneering therapies for debilitating diseases for which no satisfactory treatment exists. All our current projects use neurotrophic factors to restore function and protect neurons from further degeneration. Because neurotrophic factors cannot be delivered safely and effectively to the brain using conventional means, Ceregene has developed and is using its own proprietary technology to efficiently deliver genes for neurotrophic factors into neurons that are degenerating. Thus, rather than attempting to deliver the neurotrophic factor in an effective fashion (which decades of research suggests may be an impossible feat), treatment with our products is intended to genetically program the neurons so that they can now manufacture the neurotrophic factor at the site of degeneration. Manufactured at the site of degeneration, the neurotrophic factor should restore neuronal vitality and function, while also helping protect neurons from further degeneration. Our approach provides the means to selectively target only those regions of the brain or eye that require the neurotrophic factor, thus providing an additional level of comfort regarding safety. Additionally, a single treatment is likely to be all that is required to provide prolonged, permanent, therapeutic benefit to the patient.
By using gene delivery technology, Ceregene may be able to solve an age-old problem of how to effectively and safely deliver these potent neurotrophic factors to highly targeted sites. Given the therapeutic potential of neurotrophic factors, this approach has the potential to revolutionize the treatment of these terrible neurodegenerative and ocular diseases by both significantly reducing serious symptoms, while also protecting against further degeneration. The millions of people with Parkinson’s disease (PD), Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS), Huntington’s disease, and several ocular diseases that cause blindness (such as retinitis pigmentosa, age-related macular degeneration and glaucoma) might all benefit from Ceregene’s common approach to treating these divergent diseases with different neurotrophic factors. Clinical testing of these product candidates is ongoing in both PD and AD while candidates for the other diseases are in preclinical stages of testing and development.
How Ceregene's Gene Delivery Technology Works
Gene transfer offers an effective means of treating diseased and dying neurons with neurotrophic factors in a targeted and prolonged fashion. We have identified several neurotrophic factors to treat damaged nerve cells in several different diseases. For each disease, the gene encoding the appropriate neurotrophic factor known to provide nourishment for the key neurons that are dying is delivered to those cells in a single dosing procedure. This is accomplished through a gene therapy 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 diseasee.
To carry the neurotrophic factor genes into the targeted neurons in a safe and effective fashion, we use a harmless version of a virus that humans are commonly exposed to. This virus, called the adeno-associated virus (AAV), appears to be very safe in that it is not known to be associated with any human disease. To help provide even greater safety, we have modified AAV to assure it cannot replicate, or reproduce itself, thus assuring that the genes we deliver remain exclusively in the targeted tissue.
Our AAV-based gene delivery technology provides a broad platform to develop pioneering treatments for many different serious diseases, including Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, Huntington’s disease and several ocular diseases. We are currently employing this technology in our human clinical trials for Parkinson’s disease and Alzheimer’s disease.