VIDEO: Studies underway on ‘promising disease-modifying compounds’ for Huntington’s disease

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There are several promising disease-modifying compounds that are under study for HD. We have the advantage, if you will, in NHD in that we know the causative gene, HTT, which is located in the short arm of chromosome 4. We know, however, that the HTT gene cannot be silenced, it's necessary for embryonic development. There are efforts underway to modify the gene and/or modify the gene product. An example of one of the disease-modifying trials under study is the uniQure studies, the study drug is AMT-130. The uniQure study is a phase 1/2 randomized double-blind sham-control clinical trial that's evaluating the safety and efficacy of like I said, AMT-130, which is a microRNA interference molecule that’s delivered through a non-pathogenic viral vector directly into the striatum, so directly into the caudate and putamen, which is the area in the brain which is preferentially affected early actually in HD. So that trial is ongoing, we don’t have obviously the results of the trial, but that is an example of one of the disease-modifying Huntington protein lowering studies that is currently underway. On the symptomatic front, there is a drug under study. Sage Therapeutics is the pharmaceutical company that has this drug. It's actually a positive allosteric modulator of the NMDA receptor. And this drug is being studied in individuals with HD that have cognitive symptoms, and so there’s definitely a need for medications that address the cognitive symptoms in HD. And so, we’re hopeful that the studies are currently underway evaluating the safety and efficacy of this medication.