Uniqure presented unprecedented pivotal clinical trial data for Huntington’s disease yesterday.
It demonstrated a statistically significant 75% disease slowing of this devastating disease.
This is life-changing news for people suffering from this devastating disease for which no treatment is available today.
We expect the medicine to reach patients in the US first by late 2026.
Yesterday, the net asset value of the life sciences fund went up 11,2% and of the Genetics fund 15,9% mainly driven by the 248% share price increase of Uniqure.
The treatment consists of a one-time delivery of a gene therapy into the brain. With this medicine we expect the company to fulfill a high unmet medical need, and making billions in revenue.
Based on the positive clinical data Uniqure shared with the FDA last year it received a so-called breakthrough therapy designation and regenerative medicine advanced therapy designation, which typically shorten the timeline to approval. This disease invariably leads to a premature death, while slowly but severely damaging physical and cognitive capabilities. There is no approved treatment for Huntington’s disease.
Uniqure has several other medicine candidates of which three are being tested in humans. For two of those it has showed first encouraging clinical results. For all three medicines we expect important results across 2026.
