Alzheimer’s disease is characterized by progressive memory loss which results in severe loss of mental function and eventually death. Histological examination of the brains of patients with Alzheimer’s disease reveals amyloid plaques, neurofibillary tangles, and loss of connection between neurons. There are treatments that may delay progression of the disease, but there is no cure.
Gene therapy approaches to Alzheimer’s disease include introduction of neuroprotective genes to preserve neurons and neuronal connections, or genes to reduce the amyloid plaque burden.
To overcome the difficulty in delivering purified therapeutic proteins across the blood-brain barrier, the REGENX strategy is to use NAV® Technology to carry in the therapeutic gene to the central nervous system, allowing for therapeutic protein expression at the site where it is needed. NAV Therapeutics are significantly more efficient in delivering genes to the CNS than other rAAV serotypes, and can distribute gene expression further with each injection. The proposed therapeutic is in the preclinical stage of development.