Isis Pharmaceuticals Continues To Pursue ALS Therapy Development

Isis Pharmaceuticals is currently developing therapies for both ALS and Huntington’s disease. Their novel approach uses technology called antisense oligonucleotides (ASOs) to target and regulate specific RNAs inside neurons. Isis just completed a Phase I study of their ASO drug (ISIS SOD1Rx) in people with ALS caused by mutations in the SOD1 gene. The results of the study were positive – the ASO drug was shown to be well tolerated and safe. However, the ISIS SOD1Rx drug can only be used to treat about 2% of all ALS patients. Isis currently has plans to develop additional ALS therapies that would reach a larger population.

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