First Time Ever: Scientists Fix Huntington's Protein Defect With Ionis Pharma Drug

LONDON (Reuters) - Scientists have for the first time fixed a protein defect that causes Huntington’s disease by injecting a drug from Ionis Pharmaceuticals into the spine, offering new hope for patients with the devastating genetic disease.

The success in the early-stage clinical trial has prompted Roche to exercise its option to license the product, called IONIS-HTT(Rx), at a cost of $45 million.

Lead researcher Sarah Tabrizi, professor of clinical neurology at University College London, said the ability of the drug to tackle the underlying cause of Huntington’s by lowering levels of a toxic protein was “ground-breaking”.