What did we learn in Huntington’s disease news in September?
This month was hopeful, with encouraging results from two clinical trials for Huntington’s disease treatments, including one in Australia. We also learned of other studies, including how voice-based tools could help slow HD progression and assess treatments.
Discover what we learned about Huntington’s disease in September.
Skyhawk Therapeutics’ SKY-0515 shares promising 12-month results
Skyhawk Therapeutics reported results from Phase 1/2 of its once-daily oral treatment, SKY-0515. The trial, which took place in Australia and New Zealand, tested the safety and efficacy of the treatment, which is designed to reduce levels of huntingtin protein.
The latest results showed that, at month 15, patients who had received SKY-0515 had an improved composite Unified Huntington’s Disease Rating Scale (cUHDRS) score of an average of 0.94 points from where they began. This compared with untreated patient data from the Enroll-HD database, which declined an average of 0.65 points over the same period.
Findings also showed significant improvements in the four measures that make up the cUHDRS: daily function, movement, processing speed, and reading speed.
Although the study was small, reporting improvement at this early stage is a hopeful sign for researchers as they move toward larger trials. Read the latest press release from Skyhawk Therapeutics here.
UniQure shares four-year data from gene therapy treatment AMT-130
In a press release from September 29, 2026, UniQure shared data from its ongoing Phase 1/2 Studies of ifezuntirgene inilparvovec, also known as AMT-130. AMT-130 is a one-time gene therapy treatment, delivered through brain surgery, and designed to reduce the production of huntingtin protein.
The findings included:
- Four years after a single administration, AMT-130 continued to show meaningful signs of slowing HD progression.
- After 48 months, decline of total functional capacity (TFC) in participants demonstrated consistent slowing.
- Researchers included three high dose patients in its 36-month analysis, each with a significant impact on cUHDRS and TFC, demonstrating meaningful slowing down of HD progression.
UniQure also announced earlier this month that it is seeking approval in the US and UK for its gene therapy treatment. Huntington’s Australia CEO Lenni Duffield previously answered questions about this treatment, however, it’s still not available for trial participants in Australia.
Read the latest press release from UniQure here.
How voice-based tools can assess HD progression
A recent study published in the Journal of Neurology found that the speech index score could be used as a non-invasive, scalable tool to monitor Huntington’s disease progression and how someone responds to treatment.
Built from smartphone voice recordings, researchers matched the speech index score to clinical assessments and brain scan measurements. They also used the tool to distinguish individuals with the Huntington’s-causing mutation from those without it, with high accuracy.
The score included factors such as timing, loudness variation, and mispronunciation rate, helping to track the severity of HD, with scores increasing the more advanced it became.
As speech difficulties are a common symptom of Huntington’s disease, sometimes appearing before other motor symptoms, tools like this could be extremely useful in monitoring its progression.
Celebrating small steps of progress
September brought promising findings in Huntington’s research, including local and global research that advances our understanding of Huntington’s disease and potential treatments.
Huntington’s Australia will keep you up to date on any relevant HD news, so keep an eye on our news page and social media to stay informed.
