Will Gene Therapy Finally Provide a Cure for Huntington’s Disease?

According to a recent article published in Pharmaceutical-Technology, uniQure’s gene therapy AMT-130 may lower levels of the huntingtin protein that causes the disorder.

An abnormal gene is responsible for damage to the brain over a period of time, causing Huntington’s disease. Current treatment offers minimal relief, but the AMT-130 therapy appears to have curative potential.

About Pre-clinical Studies

Multiple animal models of Huntington’s disease were evaluated. Neurons derived from Huntington patients’ pluripotent stem cells (self- replicating) were also evaluated.

Results of these studies demonstrated AMT-130’s ability to zero in on the toxic protein. In addition, AMT-130 carries an artificial micro-RNA that silences the huntingtin gene.

About Huntington’s Disease (HD)

HD is a fatal and rare neurodegenerative disorder affecting motor function and cognitive decline. The results are complete mental and physical deterioration, generally in young adults.

HD affects about seventy thousand people in Europe and the United States. This distinguishes HD as having the greatest number of unmet needs in the entire rare disease community. As previously noted, there are currently no therapies to even slow the progression of HD.

uniQure’s Twelve-Month Follow-up Data

uniQure announced twelve-month results of the mini-pig model in February 2019. This was the largest animal model for Huntington’s disease presented to date.

The results confirmed that a one-time administration of AMT-130 caused a reduction in dose-dependency and also a reduction of the mutant protein in the brain.

Non-invasive scans demonstrated that AMT-130 led to improvement in brain cell function. It was also responsible for the partial recovery of volume in the hippocampus, a key areas of the brain that involves memory.

Therefore, delivering micro-RNAs to the brain for knockdown of the huntingtin gene has been shown to be an innovative and very promising approach to treating Huntington’s disease.

Three Companies Vying for the Market

Three companies, Wave Life Sciences, uniQure, and Roche are working on gene therapies in various phases of testing for Huntington’s disease.

Roche and its partner Ionis are developing an experimental drug called RG6042 that will limit the protein’s production. Find more about this here.

In order to be a viable candidate, Wave Life Sciences’ drug WVE-120102 had to clearly show that it was an improvement over Roche’s RG6042. Unfortunately, it’s phase 1b/2a trial failed in this regard.

Roche and uniQure are competitors with two different methods of administration. Although Roche’s drug is currently in Phase III trials, the drug will be administered frequently. On the other hand, this would not deter uniQure that is currently in early stages of development, as its therapy will only be administered once.

The fact that Huntington’s disease is controlled by a single gene (monogenic) adds weight to the positive opinions expressed by doctors in the field of neurology.


Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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