Background

Amyotrophic lateral sclerosis (ALS), often called Lou Gehrig’s disease, is a progressive nervous system disease that affects nerve cells in the brain and spinal cord, causing loss of muscle control. Most patients die within 2 to 5 years of diagnosis due to lack of effective therapies as the blood‑brain barrier (BBB) prevents entry into the brain of more than 98% of large and small-molecule drugs. Therefore, there is a requirement for ALS therapeutics that can cross the BBB and prevent disease progression.