October 2016 – Antisense Patent for the Treatment of Familial ALS

The United States Patent Office issued a key patent that describes the development of a novel therapy for a treatment of one form of familial ALS. The Center was instrumental in conceptualizing this method of treating ALS and similar ailments. The multi-year effort to demonstrate the utility of this approach was undertaken in collaboration with Ionis Pharmaceutical Corporation and the University of California, San Diego. Currently, BioGen Pharmaceuticals is conducting a Phase I study to test the safety and pharmacologic parameters of this drug in a group of ALS patients with a mutation that affects the function of an enzyme that has been associated with one form of ALS. The patent can be found here.