Alzheimer’s Disease

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.

Status

To successfully treat diseases such as Alzheimer’s disease, gene therapy needs to target the neurons within the brain. ReGenX’s AAV9 serotype is significantly more efficient in delivering genes to the CNS than other serotypes, and can distribute gene expression further with one injection. The proposed therapeutic is in the research stage of development.

Market

It is estimated that Alzheimer’s disease currently affects 2.4 million to 4.5 million Americans. The annual cost of caring for one individual with Alzheimer’s disease ranges from nearly $18,500 to more than $36,000, depending on the stage of the disease. The cost in the U.S. for caring for individuals with Alzheimer’s disease is estimated at $100 billion annually.¹ Costs for Alzheimer’s drugs average $148 to $195 per month and only 10–20 percent of patients benefit compared to placebo.²