- New gene therapy shows potential for long-lasting treatment of Huntington’s disease.
- Some patients experienced temporary inflammation, headaches, or confusion, treated as needed.
- Therapy could last a lifetime because brain cells are not naturally replaced like other tissues.
- Around 75,000 people have Huntington’s in the UK, US, and Europe; hundreds of thousands carry the mutation.
- UniQure aims to apply for a US license in early 2026, with potential launch the same year; UK and Europe discussions will follow.
- High complexity surgery and cost will limit patient access.
- Gene therapies are expensive but can be justified by long-term benefits; NHS pays £2.6m for haemophilia B treatment as an example.
Scientists are celebrating a major breakthrough in the fight against Huntington’s disease, as a new gene therapy shows promise to provide lasting effects for patients. While some participants experienced temporary inflammation that caused headaches and confusion, these side effects either resolved on their own or required steroid intervention.
Prof. Wild explained that the therapy could potentially last a lifetime because brain cells, unlike blood, bone, and skin, are not naturally replaced by the body.
Huntington’s disease affects around 75,000 people across the UK, US, and Europe, with hundreds of thousands more carrying the genetic mutation that will eventually cause the condition.
UniQure, the company behind the therapy, plans to apply for a US license in the first quarter of 2026, targeting a launch later that year. Discussions with regulators in the UK and Europe are expected to begin next year, but the initial focus will remain on the US market.
Dr. Walid Abi-Saab, UniQure’s chief medical officer, described the results as “incredibly exciting,” highlighting the therapy’s potential to fundamentally change the course of Huntington’s disease for affected families.
However, the therapy’s availability will be limited due to the complexity of the surgical procedure and the anticipated high cost. While no official price has been set, Prof. Wild noted that gene therapies are expensive, though they can be cost-effective over the long term. For comparison, the NHS covers a £2.6 million-per-patient gene therapy for haemophilia B.
