I believe this is the clinical trial they are reporting on: https://clinicaltrials.gov/study/NCT04120493
This trial also appears be open at UCSF...
I don't ask to strictly bring up politics, but instead to try and address the broad lack of understanding of how medical breakthroughs like this are made.
It's not done just by drug companies. The article says:
> UniQure says it will apply for a licence in the US in the first quarter of 2026 with the aim of launching the drug later that year.
That's true, but that doesn't talk about the tens to hundreds of research papers that have been published over likely decades to make this discovery a reality. And it doesn't talk about how much public money went into this discovery.
Many people reading this article probably have a vague idea that more than just this company was involved, but I feel it is not at all clear to the vast majority of people, since the vast majority of people are not involved in biomedical research.
I wish there was an easy way to figure out how many dollars, how many grants, how many researchers, went into achieving this breakthrough. And that the media would put that into news articles like this. Trace all the citations back a few orders, and I bet you'll find a massive number of NIH and NIHR grants.
There is unfortunately not more massive, bipartisan public outcry in the US over defunding the essential basic research the NIH does... and it's not new to the current administration, since it was attempted to be done back in 2017, too [1].
Scientists need better messaging or else we're going to stop having breakthroughs like this... and the breakthroughs are already going to slow down thanks to things like the $783 million in cuts to NIH grants that the US SCOTUS authorized in August [2].
1. https://pmc.ncbi.nlm.nih.gov/articles/PMC5468112/
2. https://www.scotusblog.com/2025/08/supreme-court-allows-trum...
Sure, but it's really sad that scientists need to justify their funding to the public - they already spend so much time justifying it to the NIH and others for funding.
So many people have had their careers jeopardized by finding pulled mid-project. I am really concerned about our research pipeline, because my post-doc friends are all applying to jobs outside the US now.
petesergeant•1h ago
> AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging our proprietary miQURE™ silencing technology. The therapeutic goal is to inhibit the production of the mutant protein (mHTT)
and the actual announcement: https://uniqure.gcs-web.com/news-releases/news-release-detai...
> 75% slowing of disease progression as measured by Unified Huntington’s Disease Rating Scale (p=0.003)
> 60% slowing of disease progression as measured by Total Functional Capacity (p=0.033)
> 88% slowing of disease progression as measured by Symbol Digit Modalities Test (p=0.057)
> 113% slowing of disease progression as measured by Stroop Word Reading Test (p=0.0021)
> 59% slowing of disease progression as measured by Total Motor Score (p=0.1741)
basisword•1h ago
CookiesOnMyDesk•1h ago
>It means the decline you would normally expect in one year would take four years after treatment, giving patients decades of "good quality life", Prof Sarah Tabrizi told BBC News.
>The first symptoms of Huntington's disease tend to appear in your 30s or 40s and is normally fatal within two decades – opening the possibility that earlier treatment could prevent symptoms from ever emerging.
didgeoridoo•46m ago
Consider that the disease typically manifests in your 30s — does this mean it would begin 4x later (and thus basically never manifest), or that your 15 year progressive decline from ~35-50 would take 4x longer (giving you a normal lifespan, albeit perhaps with some limitations in your later years)?
bjornsing•1h ago
JoshTriplett•59m ago
adcoleman6•59m ago
Sebalf•48m ago
I don't know the details of why AAV5 in particular is their vector of choice in this case, but for whatever reason thats what they've gone with. AFAIK there are no viral or other vectors that consistently infect all brain tissue when injected/ingested, so maybe that's just the best option available. Anyways, it seems that in order to get it to the actual brain tissue that is damaged by the huntington protein (all of it? One particular area?), the best way is to inject it where it needs to go. If you could just pump it into the CSF that would perhaps make things a little bit more tolerable, seeing as you could then just do a spinal tap and inject it that way, but apparently that doesn't work. Or maybe a generalized AAV5 infection has more side effect then targeted injections. Just speculating here.
saretup•46m ago
nicoburns•40m ago
tkfoss•21m ago