Chemistry: Generation 2+
IONIS-C9Rx, also known as BIIB078, is an investigational antisense medicine designed to target mutant chromosome 9 open reading frame 72 (C9ORF72) RNA. It is in development for patients with a genetic form of amyotrophic lateral sclerosis (ALS) called C9ORF72-ALS or C9-ALS, which is the most common genetic form of ALS worldwide. There is substantial evidence that this mutation is responsible for a toxic gain of function repeat expansion that can lead to rapid progressive loss of motor neurons in people with C9ORF72-ALS.
About Amyotrophic Lateral Sclerosis
ALS is a rare, fatal neurodegenerative disorder characterized by loss and dysfunction of neurons in motor pathways. It is estimated that there are ~55,000 patients diagnosed with ALS in major markets1. Approximately 90 percent of patients with ALS have no known family history, while approximately 10 percent of patients have a known genetic cause for their disease. People with ALS suffer progressive degeneration of motor neurons, which results in a declining quality of life due to muscle weakness, loss of movement and difficulty in breathing and swallowing. Currently, treatment options for patients with ALS are extremely limited with no drugs that significantly slow disease progression.