Isis, Genzyme Use Antisense to Go Where Many Drugs Fail—The Brain

Isis Pharmaceuticals is hopeful that it has identified a new way to treat ALS. Their latest device delivers RNA-silencing drugs (the antisense model that Isis is well-known for using) that are designed to shut down the RNA that enable the production of disease-causing proteins. The new delivery method (via a spinal tap) finally overcomes the long-standing challenge of getting the antisense drugs to enter the brain across the blood-brain barrier. Genzyme has an option to co-develop the treatment, which will enter clinical trials later this year, for patients with a particularly aggressive form of ALS.

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