April 15, 2021 Edited by Dr Jeff Carroll – Western Washington University
Two recent press releases from uniQure provide welcome good news: the first ever Huntington’s Disease (HD) gene therapy, known as AMT-130, has been administered via brain surgery to a small set of participants in an early safety trial. At the same time, uniQure has published findings in HD animal models that increase confidence in the drug’s ability to lower huntingtin, especially in parts of the brain most affected by HD.
The first gene therapy for HD
Gene therapy is a technique to introduce, replace, or remove genetic material from a person’s cells to treat a disease. In the case of Huntington’s disease, current gene therapies seek to inactivate the faulty message (RNA) produced by the HD gene, ultimately lowering the amount of huntingtin protein in the brain or body. There are dozens of research laboratories and companies working on different approaches to this, but uniQure’s HD gene therapy, AMT-130, is the first to be tested in humans.
We Can Never Lose HOPE….
I’m a HD advocate, Chair for HDSA San Francisco Bay Area Affiliate, an author and a blogger.