Amyotrophic Lateral Sclerosis (ALS)

ALS (also known as Lou Gehrig’s disease) is a progressive, fatal disease that causes the degeneration and death of neurons controlling voluntary muscle movement. ALS most commonly affects adults between 40 and 60 years of age, and in up to 90% of the cases is fatal within 3-5 years of onset. Approximately 5-10% of cases are inherited, with about 20% of those attributable to a specific gene mutation. ReGenX is taking an approach to treating ALS that is independent of the causative factors, by preserving neurons and neuron function through expression of a neuroprotective protein.

Status

To successfully treat diseases such as ALS, gene therapy needs to target the neurons and other cells in the central nervous system. To overcome the difficulty in delivering purified therapeutic proteins across the blood-brain barrier, the ReGenX strategy is to use AAV Vector Technology to carry in the gene for the neuroprotective protein to the central nervous system, allowing for therapeutic protein expression at the site where it is needed. AAV9 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

ALS affects as many as 30,000 in the United States, with 5,000 new cases diagnosed each year. Estimates suggest ALS is responsible for as many as five of every 100,000 deaths in people aged 20 or older.¹ Currently, there is no cure for ALS, only the symptoms can be treated. Total annual costs for the socioeconomic burden form ALS are estimated to exceed $50,000 per patient.² In advanced stages care can cost $200,000 per year.³