> 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)
Thanks for sharing this information. Do you (or anyone else here) know if these trends might be expected based on how the treatment works? For example, given than the treatment is only injected into certain parts of the brain, could we expect that some aspects of the disease will be treated better than others?
This is incredible, what a miraculous thing for sufferers and their families. Feels rare to see such a such an unquestionably good news story these days.
I think if you’re a researcher with an incredible result in a pre-print then going to the press to “own” the story before it’s been peer reviewed is fine by me.
The risk is that someone else sensationalises the story on your behalf and you don’t get to temper expectations with facts. I think the British researchers did that quite well in the BBC interview yesterday.
What part of this discovery was made thanks to NIH and/or NSF funding from the USA, or the NIHR in the UK?
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].
There's little money to be made with HD. It's a 1 in 30,000 disease. There's been little reason for anyone other than state sponsors to support its treatment. Add this to the reason's to be disgusted by capitalism. Spoken as a widower of an HD wife.
I'm sorry for your loss, I can only imagine how difficult it must feel to face a disease with so few treatment options
I just would like to say that it's not capitalism that decides if money is invested in a disease or another but just the individuals operating freely in the market. On the other hand Capitalism has been actually the main driver for the massive investments that enable the expensive research in biomedical topics.
It’s unfortunately normal that conditions with very low prevalence, receive less private investment than diseases that affect millions of people. That’s not because of a moral failing of capitalism, but a result of free market and the free decision of the population on where to allocate their resources. Imposing anything else on people would actually be the real moral failing, because what is the right allocation of resources between technological development, investments about hunger, medical development or just leisure? Let each individual decide for themselves and of course feel free to convince anyone to invest in what you consider priority.
So I think the fairest system is the one where individuals remain free to choose how to invest their time and money, while society as a whole can still decide, for example through philanthropy, to give extra support to areas some areas like rare diseases.
Said that, if you know of any organization supporting HD research that deserves any type of donation please let us know here so we can support it voluntarily.
We even do not need to calculate NIH grants; I am pretty sure that all databases that were used here are from NCBI. If there were no NIH, all research would be impossible in modern biology.
The problem is the soundbite of some of these studies on the surface is ridiculous to lay people but even good studies with bad sound bites are used as weapons against science funding in the USA. The shrimp on a treadmill study is still used as argument against science funding today.
https://www.npr.org/2011/08/23/139852035/shrimp-on-a-treadmi...
I think it is a UK study. It actually has been going on for a number of years, I've seen one of the PIs giving a number of big conference talks (Tabrizi) for a while
Medical progress has been insane in the last few years through technological breakthroughs. It's not out of reach to think that most types of cancers will be curable 20 years from now on.
The funny thing about phrases like that is that they often get repeated despite being devoid of factual content.
We should indeed keep an eye out for the defunding of medical research but as even a few quick numbers from the NIH (at least speaking for the US) show, it hasn't generally been getting less funding year over year right up to the latest numbers. All the opposite actually. Increases in funding have shrunk slightly, but that's not the same as defunding.
Similar stats apply almost globally in any relevant public funding context. After all, if there's one thing that everyone wants, from rich to poor, powerful or weak, it's to hedge against bad health and a shorter lifespan by whatever means available, and especially if those means involve throwing money that isn't even yours personally at something with publicity.. Even the most selfish politicians can usually get that through the fog of their self-interest.
This is off topic, slightly but I think a good place to say this:
I wish the media outlets would mention the fact that at least one of the scientists in this post is an immigrant in the UK. (in this case I’m not sure 1st or 2nd gen)
In the current climate of anti-immigrantion rhetoric around the world, simple things like that might help a little with the perception of immigrants as freeloaders.
There has been a strong push back against illegal-immigration in the west. The media has completely reframed the discussion to "How can they be opposed to immigration" because if they said "how can they be opposed to illegal-immigration" their argument would fall apart pretty quickly.
No one with a brain is arguing that immigration doesn't provide tremendous value.
I have a feeling this migrant didn’t get off a dinghy with all the other engineers and scientists so probably isn’t raising a lot of concern for most. Conflating “immigration is too high” with “anyone who thinks immigration is too high is a racist who thinks they are all freeloaders” doesn’t work anymore, no amount of media propaganda will change that.
This needs to be said, with Trump's cutting of College Basic Research Funds, many of these great breakthroughs will occur in other Countries. The US is/was the lead in biotech, it is now giving up its lead. That means one of the US largest industries, employing many with high pay, will shrink, due to US policies. In a few years it may not exist unless funding is restored soon. I personally know people who's grants have been cancelled due to these cuts.
But, glad to see other Countries funding their research. I wonder in face of one of the largest blunders made by the US, are they increasing funds ?
There’s another connection here, between trump and huntingtons.
Folk singer Woody Guthrie wrote the song “old man trump” about fred trump (donny’s dad), while suffering from huntingtons and living in a building owned by fred.
"If one of your parents has Huntington's disease, there's a 50% chance that you will inherit the altered gene and will eventually develop Huntington's too."
Have they never heard of genetic diagnostics? For example with a combination of preimplantation generic testing and in-vitro fertilization you can prevent passing on known genetic mutations to the next generation.
There is an ethical component here. The process you describe involves bringing new human lives into existence, then culling the embryos that carry the gene. For those of us who believe that directly ending human lives at any stage is wrong, this is problematic.
Keep in mind that this isn't a so-called "incompatible with life" genetic issue. People with the defective gene can live asymptomatically into middle age. I've lost multiple family members to HD, and have multiple living with HD today. I would give just about anything to spare them from the effects of the disease. But should we decide, moments into a human being's life, that because they carry this gene, it invalidates their entire existence?
That explains a bit more: (1) neuro-surgery introduces gene-virus to putamen and caudate nucleus; (2) virus delivers gene that produces micro-RNA; (3) the micro-RNA blocks the messenger RNA of the bad gene, reducing bad protein production.
The 2023 study is said to have 39 patients (BBC and their recent statement reports 29). The reported findings may be significant but seem small (e.g., low dose: 0.39 of 14.1 points). Earlier they reported composites from the Unified HD Rating Scale, which has the usual caveats for the behavioral and functional sub-measures (vs. the more reliable motor and cognitive). Today's statement instead focuses on the more objective measures instead of the composite.
Earlier, high-dose responders reportedly didn't just stabilize but got better -- unclear how. The more recent findings report that the disability still progressed, but slowed relative to "propensity-matched" controls. (Note 4 of 10 controls opted to join the trial after 12 months.)
Both mention improvement in NfL (neurofilament light chains), which is an objective but nonspecific (and highly variable) measure of the degree of neuronal decomposition. The statement quantifies this at ~8% -- unclear if this level is convincing.
For such an invasive treatment for a slowly progressing, relatively rare disease, they're probably gathering and publishing data as fast as possible. The short-term results seem good, and it will be good to see long-term results over time.
It's possible some effect seen is due e.g., to immune adjuvants or something else during the therapy, and I would want experts to review the propensity matching.
I would be concerned that the micro-RNA produced either also binds with epitopes from other messenger RNA or induces some immune response. Remember, there's no reversal agent or half-life elimination for such genetic treatments.
> In the UK, the NHS does pay for a £2.6m-per-patient gene therapy for haemophilia B.
A misleading data point. This group of people were treated so poorly by the state that something had to be done. I don’t think this is setting a benchmark.
One of my mom's best friends when I was a kid had Huntington's. She was a few years older than mom, and her sons were a few years older than my brother and I. One of them chose to get tested. The other chose not to. I remember thinking that was foolish, but I was seven years old. In retrospect, it's strange that a seven-year-old was privy to such things.
Huntington's is among the best candidates for a genetic cure: well known gene and mechanism, definitive pre symptomatic diagnosis, slow progression.
But I am still reluctant. It's phase 1/2 (ie exploratory) and the phase 3 is the hard part that takes many years. Also it's disease slowing not stopping.
Combine that with the fact that it's gene therapy delivered via neurosurgery, and you're looking at a major leap in both biotech and clinical application
My husband has Parkinson’s disease, adding PD-5 herbal formula to his nighttime Parkinson’s meds has completely changed his sleep issues. He slept all day and up all night, we had to hire care nurses. Now using this PD-5 medicine for the last four months and a normal routine he sleeps almost completely through the night and may get up once to use the restroom. It’s improved so much we were able to let go of the night nurses. This medicine also helps a ton with memory. we got the treatment from www. Limitlesshealthcenter. com I am absolutely confident that this program offers a viable solution. I hope someone find this helpful, We feel very fortunate to have learned about pd-5.
39 comments
[ 3.7 ms ] story [ 62.3 ms ] thread> 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)
And here my government is actively working to suppress mRNA therapies because of fucking politics. Fuck them.
The risk is that someone else sensationalises the story on your behalf and you don’t get to temper expectations with facts. I think the British researchers did that quite well in the BBC interview yesterday.
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...
I just would like to say that it's not capitalism that decides if money is invested in a disease or another but just the individuals operating freely in the market. On the other hand Capitalism has been actually the main driver for the massive investments that enable the expensive research in biomedical topics.
It’s unfortunately normal that conditions with very low prevalence, receive less private investment than diseases that affect millions of people. That’s not because of a moral failing of capitalism, but a result of free market and the free decision of the population on where to allocate their resources. Imposing anything else on people would actually be the real moral failing, because what is the right allocation of resources between technological development, investments about hunger, medical development or just leisure? Let each individual decide for themselves and of course feel free to convince anyone to invest in what you consider priority.
So I think the fairest system is the one where individuals remain free to choose how to invest their time and money, while society as a whole can still decide, for example through philanthropy, to give extra support to areas some areas like rare diseases.
Said that, if you know of any organization supporting HD research that deserves any type of donation please let us know here so we can support it voluntarily.
We should indeed keep an eye out for the defunding of medical research but as even a few quick numbers from the NIH (at least speaking for the US) show, it hasn't generally been getting less funding year over year right up to the latest numbers. All the opposite actually. Increases in funding have shrunk slightly, but that's not the same as defunding.
Similar stats apply almost globally in any relevant public funding context. After all, if there's one thing that everyone wants, from rich to poor, powerful or weak, it's to hedge against bad health and a shorter lifespan by whatever means available, and especially if those means involve throwing money that isn't even yours personally at something with publicity.. Even the most selfish politicians can usually get that through the fog of their self-interest.
https://report.nih.gov/funding/categorical-spending#/
I wish the media outlets would mention the fact that at least one of the scientists in this post is an immigrant in the UK. (in this case I’m not sure 1st or 2nd gen)
In the current climate of anti-immigrantion rhetoric around the world, simple things like that might help a little with the perception of immigrants as freeloaders.
Just a thought.
There has been a strong push back against illegal-immigration in the west. The media has completely reframed the discussion to "How can they be opposed to immigration" because if they said "how can they be opposed to illegal-immigration" their argument would fall apart pretty quickly.
No one with a brain is arguing that immigration doesn't provide tremendous value.
But, glad to see other Countries funding their research. I wonder in face of one of the largest blunders made by the US, are they increasing funds ?
Good science is not political. Politicians making it so are idiots at best and evil at worst. See also "Hanlon's Razor"
Folk singer Woody Guthrie wrote the song “old man trump” about fred trump (donny’s dad), while suffering from huntingtons and living in a building owned by fred.
Have they never heard of genetic diagnostics? For example with a combination of preimplantation generic testing and in-vitro fertilization you can prevent passing on known genetic mutations to the next generation.
Keep in mind that this isn't a so-called "incompatible with life" genetic issue. People with the defective gene can live asymptomatically into middle age. I've lost multiple family members to HD, and have multiple living with HD today. I would give just about anything to spare them from the effects of the disease. But should we decide, moments into a human being's life, that because they carry this gene, it invalidates their entire existence?
This is promising but needs publication and expert review.
Here's the actual company statement from today:
There's also a June 2024 article: That explains a bit more: (1) neuro-surgery introduces gene-virus to putamen and caudate nucleus; (2) virus delivers gene that produces micro-RNA; (3) the micro-RNA blocks the messenger RNA of the bad gene, reducing bad protein production.The 2023 study is said to have 39 patients (BBC and their recent statement reports 29). The reported findings may be significant but seem small (e.g., low dose: 0.39 of 14.1 points). Earlier they reported composites from the Unified HD Rating Scale, which has the usual caveats for the behavioral and functional sub-measures (vs. the more reliable motor and cognitive). Today's statement instead focuses on the more objective measures instead of the composite.
Earlier, high-dose responders reportedly didn't just stabilize but got better -- unclear how. The more recent findings report that the disability still progressed, but slowed relative to "propensity-matched" controls. (Note 4 of 10 controls opted to join the trial after 12 months.)
Both mention improvement in NfL (neurofilament light chains), which is an objective but nonspecific (and highly variable) measure of the degree of neuronal decomposition. The statement quantifies this at ~8% -- unclear if this level is convincing.
For such an invasive treatment for a slowly progressing, relatively rare disease, they're probably gathering and publishing data as fast as possible. The short-term results seem good, and it will be good to see long-term results over time.
It's possible some effect seen is due e.g., to immune adjuvants or something else during the therapy, and I would want experts to review the propensity matching.
I would be concerned that the micro-RNA produced either also binds with epitopes from other messenger RNA or induces some immune response. Remember, there's no reversal agent or half-life elimination for such genetic treatments.
So: room for hope but also for caution.
A misleading data point. This group of people were treated so poorly by the state that something had to be done. I don’t think this is setting a benchmark.
https://haemophilia.org.uk/public-inquiry/the-infected-blood...
But I am still reluctant. It's phase 1/2 (ie exploratory) and the phase 3 is the hard part that takes many years. Also it's disease slowing not stopping.