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IONIS-SOD1Rx

Biogen, Inc.

Drug Names(s): ISIS 333611, ISIS-BIIB3Rx, ISIS-BIIB3 Rx, BIIB067, ISIS-SOD1Rx, BIIB 067, BIIB-067

Description: ISIS-SOD1Rx is an antisense drug designed to inhibit the production of SOD1. Mutations in the gene for SOD1 have been associated with about 20% of cases of familial ALS. Since this enzyme protects the body against the build up of a free radical, one theory had been that ALS was caused by the build up of these toxic molecules. However, a more current theory is that a gain in function defect disrupts cellular functions.

Because antisense drugs do not cross the blood-brain barrier, a small pump administers the drug directly into the central nervous system infusing the drug into the cerebral spinal fluid.

Deal Structure: ISIS-SOD1Rx is being developed by Isis Pharmaceuticals in conjunction with funding from the ALS Association and the Muscular Dystrophy Association.

Isis and Biogen Idec
In September 2013, Biogen Idec and Isis announced that the companies have entered into a broad, multi-year collaboration to leverage antisense technology to advance the treatment of neurological diseases.

As part of the six-year research collaboration, Biogen Idec will gain exclusive rights to the use of Isis' antisense technology to develop therapies for neurological diseases. Terms of this collaboration include: