Our DNA makes us who we are and contains the basic roadmap for how we will grow and develop. In each cell, DNA serves as the command center providing instructions for proteins and cells to grow, mature, divide or die. If there is a mistake or change in the DNA, the command center sends the wrong message. When these errors occur in brain cells, they can lead to debilitating neurological and neuromuscular diseases like amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), Parkinson’s disease and Alzheimer’s disease – some of the most difficult conditions to treat.
To meet these challenges, Biogen scientists have been collaborating with Ionis Pharmaceuticals to develop ASOs (antisense oligonucleotides) that may provide hope to patients in need.
ASOs are the same types of building blocks that make up DNA and RNA, the protein-making machinery in the cell.1,2 ASOs bind and alter the cell’s RNA and as a consequence, modify, reduce or restore important proteins.3
“When we looked at the [ASO] data, I literally got chills,” says Toby Ferguson, M.D., Ph.D., Vice President of Biogen’s Neuromuscular Development Unit, a former practicing neurologist who saw every day the incredible need for a breakthrough. “I realized that what we’re developing could possibly change the future for patients with neurological diseases.”