Uniqure 76% up on significant improvement in Huntington disease

Yesterday, portfolio company Uniqure saw its share price surge 76%. For the first time in history, a company has demonstrated an effective treatment for Huntington’s disease. The gene therapy slowed down the disease by 80%.

About Huntington’s

Huntington’s disease is an incurable disease of the central nervous system that is mostly inherited. The earliest symptoms are often subtle problems with mood or mental/psychiatric abilities. It also causes dementia and hyperkinetic movements.

Next steps

The positive clinical trial results have triggered the FDA to allow the company to discuss a potential fast-track approval, to bring the treatment to patients as soon as possible. The outcome of this discussion is expected later in 2024.

Uniqure

Uniqure is a gene therapy company that has one product on the market, for the treatment of hemophilia-B (inlicensed by CSL).

Later this year, it will have 4 medicine candidates in clinical testing, also for ALS, epilepsy, and Fabry disease. Potential treatments for Alzheimer’s and another approach to tackle ALS are still in preclinical development.

Prior to the news, Uniqure made up 2% of the Aescap Life Sciences fund and 5% of Aescap Genetics. As presented at our Annual Meeting, the company is one of the extremely undervalued in the funds.