Last week, Rare Disease Report
sat down with George Yohrling, PhD, the Senior Director, Mission and Scientific Affairs for the Huntington’s Disease Society of America (HDSA).
In addition to a conversation about immunotherapies in Huntington’s disease
also spoke with Yohrling about AMT-130, uniQure N.V.’s gene therapy which received an Orphan Drug Designation
: Can you briefly explain the way that AMT-130 operates?
The AMT-130 is a gene therapy, and it’s a Huntingtin-lowering approach that would be mediated through viral delivery of adeno-associated virus, or AAV. In the case of uniQure, they use AAV-5, which will deliver a micro-RNA, and this micro-RNA is directed to bind to the RNA, or the message, of the Huntingtin protein. The end result would be the elimination of that RNA, resulting in a decrease of the Huntingtin protein being produced by the body.
: Is uniQure alone in trying to treat HD in this way?
uniQure is one of several companies working in this space, with a viral mediated, or delivery, approach; Genzyme-Sanofi is working in collaboration with Voyager. Spark Therapeutics in Philadelphia is, too, and all are taking similar approaches with slightly different AAV-serotypes. The idea is that direct injection of these viruses into the brain – probably the cortex or striatum – of HD patients. The idea is that once these viruses are injected, the brain would start producing the silencing therapy – the micro-RNA – forever. We suspect that this would be a one-time treatment.
: Since there are other companies, like Genzyme-Sanofi and Spark, working on gene therapies for HD, what makes uniQure’s approach stand out?
Don’t hold me to this, but it sounds like uniQure is rapidly approaching the clinic. What makes AMT-130 unique is that if the company is first to the clinic, they will be the first gene therapy in the clinic for Huntington’s disease.
In your opinion, do you think that AMT-130 could be a potential cure for HD?
I hate using the word “cure,” but this could certainly be disease-modifying. I think that’s the hope of all of these Huntingtin-lowering approaches. Whether it’s these gene therapy approaches, or it’s the antisense oligonucleotides, the assumed result is that (patients) would produce less of the toxic protein that causes the disease. Hopefully, and we don’t know this yet, but, hopefully, it can be disease-altering.
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For more information about Huntington’s disease and the work of the Huntington’s Disease Society of America, please visit HDSA.org