Ionis Pharma Offers New Hope for Huntington's Disease

Dec 11, 2017


According to a Reuters article, scientists have fixed a protein defect that causes Huntington’s disease by injecting a drug from Ionis Pharmaceuticals into the spine.

After success in an early-stage clinical trial, Roche will exercise its option to license the product, called IONIS-HTT(Rx), at a cost of $45 million, the story said.

Frank Bennett, Ionis senior vice president of research, said the reported protein reductions exceeded their expectations, but experts warned that the results were still in early stages.

There is currently no disease-modifying treatment for the condition, and existing drugs only manage symptoms.

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