HD Clinical Trials

Gene Therapy AMT-130 for Huntington’s Disease

The Huntington’s disease (HD) community has never been so hopeful that a therapy will be found for HD in the next few years.

A new development in gene therapy was announced by uniQure, whose focus is to advance the future of medicine through gene therapy. http://www.uniqure.com/about/corporate-mission.php

“The U.S. Food and Drug Administration (FDA) has completed its review of our Investigational New Drug (IND) application for AMT-130, allowing us to begin our planned Phase I/II study.  We expect that in the second half of 2019 AMT-130 will be the first ONE-TIME administered AAV gene therapy to enter clinical testing for the treatment of Huntington’s disease.”  To read about the clinical trial, visit http://uniqure.com/gene-therapy/huntingtons-disease.php

uniQure’s gene therapy candidate for Huntington’s disease is differentiated in that:

  • AMT-130 targets the deep brain structures known for the disease pathology onset.
  • AMT-130 silences mutant huntingtin protein at levels not demonstrated in other studies.
  • AMT-130 targets the accumulation of the exon 1 HTT fragment, the most toxic source of abnormal protein aggregation in Huntington’s disease.

The AMT-130 trial will be based at HD clinical sites in the Unites States. It’s not known at this time what sites or how many, yet. These will be publicly announced when they come online. UniQure hopes to begin enrolling patients before the end of 2019.  It is brain surgery.  https://en.hdbuzz.net/274

Please sign up for EnrollHD if you would like to participate in a HD clinical trial study.  https://www.enroll-hd.org/

We Can Never Lose HOPE….. 




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