• Willeke M C van Roon-Mom, Roos Raymund A C RAC 2 Department of Neurology, Leiden University Medical Center , Leiden, the Netherlands ., and Susanne T de Bot.
• 1 Department of Human Genetics, Leiden University Medical Center , Leiden, the Netherlands .
• Nucleic Acid Ther. 2018 Apr 1; 28 (2): 59-62.

AbstractOn December 11 of 2017, Ionis Pharmaceuticals published a press release announcing dose-dependent reductions of mutant huntingtin protein in their HTTRx Phase 1/2a study in Huntington disease (HD) patients. The results from this Ionis trial have gained much attention from the patient community and the oligonucleotide therapeutics field, since it is the first trial targeting the cause of HD, namely the mutant huntingtin protein, using antisense oligonucleotides (ASOs). The press release also states that the primary endpoints of the study (safety and tolerability) were met, but does not contain data. This news follows the approval of another therapeutic ASO nusinersen (trade name Spinraza) for a neurological disease, spinal muscular atrophy, by the U.S. Food and Drug Administration and European Medicines Agency, in 2016 and 2017, respectively. Combined, this offers hope for the development of the HTTRx therapy for HD patients.

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