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IONIS-C9

Generation 2+ antisense drug

IONIS-C9Rx also known as BIIB078, is a Generation 2+ antisense drug designed to reduce the production of the mutated chromosome 9 open reading frame 72 (C9ORF72) gene. A mutation in the C9ORF72 gene results in an inherited form of amyotrophic lateral sclerosis (ALS), referred to as C9ORF72-ALS, the leading known genetic cause 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. C9ORF72-ALS is a fatal disease characterized by muscle weakness, loss of movement, difficulty in breathing and swallowing.

About Amyotrophic Lateral Sclerosis

ALS is a rare, fatal neurodegenerative disorder characterized by loss and dysfunction of neurons in motor pathways with a prevalence of approximately five cases per 100,000 persons in the U.S.1 People with ALS suffer progressive degeneration of the motor neurons, which results in a declining quality of life and ultimately death. A mutation in the C9ORF72 gene results in an inherited form of ALS, referred to as C9ORF72-ALS. Familial ALS represents approximately 15 to 20 percent of all cases of ALS and C9ORF72-related ALS accounts for up to 34 percent of all familial cases.2,3 It is currently the leading known genetic cause of ALS worldwide. People with C9ORF72-ALS experience muscle weakness, loss of movement, difficulty in breathing and swallowing and eventually succumb to their disease. Currently, treatment options for patients with ALS are extremely limited with no drugs that significantly slow disease progression.

Tofersen

Generation 2+ antisense drug

Tofersen (formerly IONIS-SOD1Rx), also known as BIIB067, is an antisense drug designed to reduce the production of superoxide dismutase 1 (SOD1), which is the best understood genetic cause of familial amyotrophic lateral sclerosis (ALS). ALS is a rare, fatal neurodegenerative disorder. Patients with ALS suffer progressive degeneration of the motor neurons, which results in a declining quality of life and ultimately death. A mutation in the SOD-1 gene results in an inherited form of ALS, referred to as SOD1-ALS. There is substantial evidence that mutations in the SOD1 gene are responsible for a toxic gain of function that can lead to rapid progressive loss of motor neurons in patients with SOD1-ALS. As a result, patients with SOD1-ALS experience muscle weakness, loss of movement, difficulty in breathing and swallowing and eventually succumb to their disease. Currently, treatment options for patients with ALS are extremely limited with no drugs currently available that significantly slow disease progression.

About Amyotrophic Lateral Sclerosis

ALS is a rare, fatal neurodegenerative disorder. Patients with ALS suffer progressive degeneration of the motor neurons, which results in a declining quality of life and ultimately death. There is substantial evidence that mutations in the SOD1 gene are responsible for a toxic gain of function that can lead to progressive loss of motor neurons in patients with SOD1-ALS. As a result, patients with SOD1-ALS experience muscle weakness, loss of movement, difficulty in breathing and swallowing and eventually succumb to their disease. SOD1-ALS is the second most common familial form of ALS, accounting for up to 20 percent of familial ALS. Familial ALS represents approximately 15 to 20 percent of all cases of ALS. Currently, treatment options for patients with ALS are extremely limited with no drugs that significantly slow disease progression.