UniQure spent years designing an ethical trial - hand in hand with the FDA - for an accelerated approval process relying on natural history data as a control (vs a placebo group having sham brain surgery and potentially missing their own window for treatment as the disease progressed).
November 16, 2025 at 4:23 PM
UniQure spent years designing an ethical trial - hand in hand with the FDA - for an accelerated approval process relying on natural history data as a control (vs a placebo group having sham brain surgery and potentially missing their own window for treatment as the disease progressed).
So most people facing it have already watched it play out across their families, across generations. They’ve been care partners for every step of it, they know what’s ahead.
The UniQure trial news two months ago was some of the most promising news the community has ever had—but now it’s in limbo.
The UniQure trial news two months ago was some of the most promising news the community has ever had—but now it’s in limbo.
November 16, 2025 at 4:23 PM
So most people facing it have already watched it play out across their families, across generations. They’ve been care partners for every step of it, they know what’s ahead.
The UniQure trial news two months ago was some of the most promising news the community has ever had—but now it’s in limbo.
The UniQure trial news two months ago was some of the most promising news the community has ever had—but now it’s in limbo.
I don’t expect a petition to change anything directly (even if the FDA was working like it used to).
But I’m sharing it - and will, again and again - because the good news about the UniQure trial broke containment and this news hasn’t. Because it’s a huge show of support for folks who need it.
But I’m sharing it - and will, again and again - because the good news about the UniQure trial broke containment and this news hasn’t. Because it’s a huge show of support for folks who need it.
November 16, 2025 at 4:23 PM
I don’t expect a petition to change anything directly (even if the FDA was working like it used to).
But I’m sharing it - and will, again and again - because the good news about the UniQure trial broke containment and this news hasn’t. Because it’s a huge show of support for folks who need it.
But I’m sharing it - and will, again and again - because the good news about the UniQure trial broke containment and this news hasn’t. Because it’s a huge show of support for folks who need it.
Last week, the FDA decided to change its mind. It’s threatening to reject the trial design they’ve been in alignment on for years- seemingly part of the broader attacks on vaccines and other clinical advances that don’t value double-blind placebo testing over reality.
en.hdbuzz.net/uniqure-and-...
en.hdbuzz.net/uniqure-and-...
UniQure and FDA No Longer in Alignment on Approval Pathway for AMT-130 – HDBuzz
en.hdbuzz.net
November 16, 2025 at 4:23 PM
Last week, the FDA decided to change its mind. It’s threatening to reject the trial design they’ve been in alignment on for years- seemingly part of the broader attacks on vaccines and other clinical advances that don’t value double-blind placebo testing over reality.
en.hdbuzz.net/uniqure-and-...
en.hdbuzz.net/uniqure-and-...
The AMT-260 was something I only heard about last week at a uniQure panel at the #EpilepsyAwarenessDayDisneyland expo in Anaheim.
www.uniqure.com/programs-pip...
www.uniqure.com/programs-pip...
Temporal Lobe Epilepsy (TLE) | Programs & Pipeline
Reimagining the future of healthcare through the power of gene therapy.
www.uniqure.com
November 18, 2025 at 2:06 AM
The AMT-260 was something I only heard about last week at a uniQure panel at the #EpilepsyAwarenessDayDisneyland expo in Anaheim.
www.uniqure.com/programs-pip...
www.uniqure.com/programs-pip...
Fast forward to now, when we learn that things aren’t exactly on track for the Huntington’s “cure”
You’ll remember how the share price jumped on the news reports and now here’s the reality check: the stock is down to where it should be, but should sink lower📉
en.hdbuzz.net/uniqure-and-...?
You’ll remember how the share price jumped on the news reports and now here’s the reality check: the stock is down to where it should be, but should sink lower📉
en.hdbuzz.net/uniqure-and-...?
November 10, 2025 at 6:37 PM
Fast forward to now, when we learn that things aren’t exactly on track for the Huntington’s “cure”
You’ll remember how the share price jumped on the news reports and now here’s the reality check: the stock is down to where it should be, but should sink lower📉
en.hdbuzz.net/uniqure-and-...?
You’ll remember how the share price jumped on the news reports and now here’s the reality check: the stock is down to where it should be, but should sink lower📉
en.hdbuzz.net/uniqure-and-...?
November 5, 2025 at 10:55 AM
@amyklobuchar.com If the FDA rejects the Uniqure application for a biologics license for a Huntington’s treatment after both the last administration and this indicated no concerns with their research protocol that would be arbitrary and capricious. Please investigate!
November 7, 2025 at 5:43 PM
@amyklobuchar.com If the FDA rejects the Uniqure application for a biologics license for a Huntington’s treatment after both the last administration and this indicated no concerns with their research protocol that would be arbitrary and capricious. Please investigate!
Moving goalposts. The new Prasad regime.
FDA submission for UniQure Huntington's drug thrown into question
The company said the agency had changed its position on what level of data were sufficient for it to apply for approval for its gene therapy.
www.statnews.com/2025/11/03/h...
FDA submission for UniQure Huntington's drug thrown into question
The company said the agency had changed its position on what level of data were sufficient for it to apply for approval for its gene therapy.
www.statnews.com/2025/11/03/h...
UniQure's FDA submission for its Huntington's disease therapy thrown into question
In a major setback, Uniqure said that the timing of when it can file its Huntington's treatment for FDA approval "is now unclear."
www.statnews.com
November 4, 2025 at 1:59 AM
Moving goalposts. The new Prasad regime.
FDA submission for UniQure Huntington's drug thrown into question
The company said the agency had changed its position on what level of data were sufficient for it to apply for approval for its gene therapy.
www.statnews.com/2025/11/03/h...
FDA submission for UniQure Huntington's drug thrown into question
The company said the agency had changed its position on what level of data were sufficient for it to apply for approval for its gene therapy.
www.statnews.com/2025/11/03/h...
for building mutant huntingtin. This results in lower levels of mutant huntingtin in the brain. Results from the trial, involving 29 patients treated in the UK and US, have been released by the company uniQure, although the full details are yet to be published. Three years after treatment, those on
October 19, 2025 at 4:08 PM
for building mutant huntingtin. This results in lower levels of mutant huntingtin in the brain. Results from the trial, involving 29 patients treated in the UK and US, have been released by the company uniQure, although the full details are yet to be published. Three years after treatment, those on
Results from the trial have been released in a statement by the company uniQure. The data from the company HAS been shared with independent scientists for review to be published.
• www.bbc.com/news/article...
• www.globenewswire.com/news-release...
🧪🧵⬇️
• www.bbc.com/news/article...
• www.globenewswire.com/news-release...
🧪🧵⬇️
October 19, 2025 at 4:03 PM
Results from the trial have been released in a statement by the company uniQure. The data from the company HAS been shared with independent scientists for review to be published.
• www.bbc.com/news/article...
• www.globenewswire.com/news-release...
🧪🧵⬇️
• www.bbc.com/news/article...
• www.globenewswire.com/news-release...
🧪🧵⬇️
🤔However, while the results have been released in a statement by the company uniQure, they have not yet been published in full for review by other specialists, so stay tuned.
www.bbc.co.uk/news/article...
www.bbc.co.uk/news/article...
Huntington's disease successfully treated for first time
One of the most devastating diseases finally has a treatment that can slow its progression and transform lives, tearful doctors tell BBC.
www.bbc.co.uk
September 29, 2025 at 9:41 AM
🤔However, while the results have been released in a statement by the company uniQure, they have not yet been published in full for review by other specialists, so stay tuned.
www.bbc.co.uk/news/article...
www.bbc.co.uk/news/article...
In a first, a gene therapy seems to slow Huntington disease
Small study suggests uniQure drug could be first successful treatment for devastating brain disorder
BYJOCELYN KAISER
🧪 www.science.org/content/arti...
Small study suggests uniQure drug could be first successful treatment for devastating brain disorder
BYJOCELYN KAISER
🧪 www.science.org/content/arti...
In a first, a gene therapy seems to slow Huntington disease
Small study suggests uniQure drug could be first successful treatment for devastating brain disorder
www.science.org
September 26, 2025 at 4:16 AM
In a first, a gene therapy seems to slow Huntington disease
Small study suggests uniQure drug could be first successful treatment for devastating brain disorder
BYJOCELYN KAISER
🧪 www.science.org/content/arti...
Small study suggests uniQure drug could be first successful treatment for devastating brain disorder
BYJOCELYN KAISER
🧪 www.science.org/content/arti...
The Huntington's disease treatment is expected to cost $2-5m.
Its owners uniQure expect to rake in tens of billions in profit from treating hundreds of thousands of patients.
People celebrate the Huntingdon's disease advance, but never ask why it costs 100x what it should.
Its owners uniQure expect to rake in tens of billions in profit from treating hundreds of thousands of patients.
People celebrate the Huntingdon's disease advance, but never ask why it costs 100x what it should.
September 25, 2025 at 11:12 AM
The Huntington's disease treatment is expected to cost $2-5m.
Its owners uniQure expect to rake in tens of billions in profit from treating hundreds of thousands of patients.
People celebrate the Huntingdon's disease advance, but never ask why it costs 100x what it should.
Its owners uniQure expect to rake in tens of billions in profit from treating hundreds of thousands of patients.
People celebrate the Huntingdon's disease advance, but never ask why it costs 100x what it should.
uniQure press release rather than peer reviewed publication, but still, very exciting
“Three years after surgery there was an average 75% slowing of the disease based on a measure which combines cognition, motor function and the ability to manage in daily life”
www.bbc.co.uk/news/article...
“Three years after surgery there was an average 75% slowing of the disease based on a measure which combines cognition, motor function and the ability to manage in daily life”
www.bbc.co.uk/news/article...
Huntington's disease successfully treated for first time
One of the most devastating diseases finally has a treatment that can slow its progression and transform lives, tearful doctors tell BBC.
www.bbc.co.uk
September 25, 2025 at 5:58 AM
uniQure press release rather than peer reviewed publication, but still, very exciting
“Three years after surgery there was an average 75% slowing of the disease based on a measure which combines cognition, motor function and the ability to manage in daily life”
www.bbc.co.uk/news/article...
“Three years after surgery there was an average 75% slowing of the disease based on a measure which combines cognition, motor function and the ability to manage in daily life”
www.bbc.co.uk/news/article...
Researchers announced that a one time gene therapy, AMT-130 (developed by UniQure), slowed the progression of Huntington’s Disease by ~75% over three years.
Until now, no therapy could alter its course. AMT-130, delivered directly into the brain, silences the faulty gene driving the disease.
Until now, no therapy could alter its course. AMT-130, delivered directly into the brain, silences the faulty gene driving the disease.
September 25, 2025 at 9:49 AM
Researchers announced that a one time gene therapy, AMT-130 (developed by UniQure), slowed the progression of Huntington’s Disease by ~75% over three years.
Until now, no therapy could alter its course. AMT-130, delivered directly into the brain, silences the faulty gene driving the disease.
Until now, no therapy could alter its course. AMT-130, delivered directly into the brain, silences the faulty gene driving the disease.
uniQure company reports gene therapy clinical trial results showing that miRNA-like silencing of the huntingtin gene slowed progression of the Huntington’s disease by 75% after three years.
The miRNA is delivered by AAV via a neurosurgery.
Amazing.
I don't hold their stocks...
The miRNA is delivered by AAV via a neurosurgery.
Amazing.
I don't hold their stocks...
September 24, 2025 at 7:51 PM
uniQure company reports gene therapy clinical trial results showing that miRNA-like silencing of the huntingtin gene slowed progression of the Huntington’s disease by 75% after three years.
The miRNA is delivered by AAV via a neurosurgery.
Amazing.
I don't hold their stocks...
The miRNA is delivered by AAV via a neurosurgery.
Amazing.
I don't hold their stocks...
An experimental gene therapy from Uniqure slowed the progression of Huntington’s disease by 75% after three years
www.statnews.com/2025/09/24/h...
www.statnews.com/2025/09/24/h...
Experimental gene therapy for Huntington’s markedly slowed disease progression in key trial
An experimental gene therapy slowed the progression of Huntington’s disease by 75% after three years — study results likely to support the first approval of a genetic treatment for the condition.
www.statnews.com
September 24, 2025 at 5:49 PM
An experimental gene therapy from Uniqure slowed the progression of Huntington’s disease by 75% after three years
www.statnews.com/2025/09/24/h...
www.statnews.com/2025/09/24/h...
🔺 NEW: Huntington’s disease is treated successfully for the first time
After early clinical trials, researchers in the UK say a new gene therapy developed by uniQure which is delivered by brain surgery worked ‘better than we dreamt’
After early clinical trials, researchers in the UK say a new gene therapy developed by uniQure which is delivered by brain surgery worked ‘better than we dreamt’
Huntington’s disease is treated successfully for the first time
After early clinical trials, researchers in the UK say a new gene therapy developed by uniQure which is delivered by brain surgery worked ‘better than we dreamt’
www.thetimes.com
September 24, 2025 at 5:23 PM
🔺 NEW: Huntington’s disease is treated successfully for the first time
After early clinical trials, researchers in the UK say a new gene therapy developed by uniQure which is delivered by brain surgery worked ‘better than we dreamt’
After early clinical trials, researchers in the UK say a new gene therapy developed by uniQure which is delivered by brain surgery worked ‘better than we dreamt’
This is amazing! The company behind it is uniQure and it's a type of gene therapy. Does anyone know the story about the preclinical work leading up to it? It know a lot about the discovery of the HTT mutation (an amazing story!). I'm curious about this specific microRNA approach: who developed it?
September 24, 2025 at 4:16 PM
This is amazing! The company behind it is uniQure and it's a type of gene therapy. Does anyone know the story about the preclinical work leading up to it? It know a lot about the discovery of the HTT mutation (an amazing story!). I'm curious about this specific microRNA approach: who developed it?
Should be, as it was invented in Britain.
Good idea by uniQure, as they are protected from the meddling of the Republicans and their weirdo ideas.
Maybe we will see more US medical companies teaming up with European universities to do the same.
Good idea by uniQure, as they are protected from the meddling of the Republicans and their weirdo ideas.
Maybe we will see more US medical companies teaming up with European universities to do the same.
September 24, 2025 at 4:02 PM
Should be, as it was invented in Britain.
Good idea by uniQure, as they are protected from the meddling of the Republicans and their weirdo ideas.
Maybe we will see more US medical companies teaming up with European universities to do the same.
Good idea by uniQure, as they are protected from the meddling of the Republicans and their weirdo ideas.
Maybe we will see more US medical companies teaming up with European universities to do the same.
the UCL writeup is more technical - uniQure treatment called AMT-130
www.ucl.ac.uk/news/2025/se...
www.ucl.ac.uk/news/2025/se...
Gene therapy appears to slow Huntington’s disease progression
A global clinical trial for a new Huntington’s disease treatment has posted positive results today, announced by trial sponsor uniQure and UCL scientists.
www.ucl.ac.uk
September 24, 2025 at 3:58 PM
the UCL writeup is more technical - uniQure treatment called AMT-130
www.ucl.ac.uk/news/2025/se...
www.ucl.ac.uk/news/2025/se...
Thoughts on this? >> uniQure Announces Appointment of Kylie O’Keefe as Chief
Customer and Strategy Officer - GlobeNewswire >> Comment below! #strategy #competitiveintelligence #marketing #biotech #pharmaceutical #competitivemarketing #healthcare #pharma
Customer and Strategy Officer - GlobeNewswire >> Comment below! #strategy #competitiveintelligence #marketing #biotech #pharmaceutical #competitivemarketing #healthcare #pharma
uniQure Announces Appointment of Kylie O’Keefe as Chief
Customer and Strategy Officer - GlobeNewswire
uniQure Announces Appointment of Kylie O’Keefe as Chief Customer and Strategy Officer GlobeNewswire
dlvr.it
June 11, 2025 at 1:30 PM
Thoughts on this? >> uniQure Announces Appointment of Kylie O’Keefe as Chief
Customer and Strategy Officer - GlobeNewswire >> Comment below! #strategy #competitiveintelligence #marketing #biotech #pharmaceutical #competitivemarketing #healthcare #pharma
Customer and Strategy Officer - GlobeNewswire >> Comment below! #strategy #competitiveintelligence #marketing #biotech #pharmaceutical #competitivemarketing #healthcare #pharma
#uniQure $QURE update:
AMT-130: FDA CMC, stat analysis meetings held; will report on outcome once meeting minutes received; 3rd cohort (with immunosuppression) safety consistent w/ 1st 2 (also Nfl), but IS has its own side effects
$400M cash-->H2 2027
finance.yahoo.com/news/uniqure...
AMT-130: FDA CMC, stat analysis meetings held; will report on outcome once meeting minutes received; 3rd cohort (with immunosuppression) safety consistent w/ 1st 2 (also Nfl), but IS has its own side effects
$400M cash-->H2 2027
finance.yahoo.com/news/uniqure...
uniQure Announces First Quarter 2025 Financial Results and Highlights of Recent Company Progress
~ AMT-130 granted Breakthrough Therapy designation by FDA ~ ~ Initial safety data from third cohort of Phase I/II study show AMT-130 continues to be generally well-tolerated, with no treatment-related...
finance.yahoo.com
May 9, 2025 at 11:19 AM
#uniQure $QURE update:
AMT-130: FDA CMC, stat analysis meetings held; will report on outcome once meeting minutes received; 3rd cohort (with immunosuppression) safety consistent w/ 1st 2 (also Nfl), but IS has its own side effects
$400M cash-->H2 2027
finance.yahoo.com/news/uniqure...
AMT-130: FDA CMC, stat analysis meetings held; will report on outcome once meeting minutes received; 3rd cohort (with immunosuppression) safety consistent w/ 1st 2 (also Nfl), but IS has its own side effects
$400M cash-->H2 2027
finance.yahoo.com/news/uniqure...
Uniqure has been testing this at a site in the US as well. They had accelerated approval for it, and are now going to meet with the FDA and then hopefully file for a biologics license. Hopefully, administration chaos won’t delay consideration.
September 24, 2025 at 3:51 PM
Uniqure has been testing this at a site in the US as well. They had accelerated approval for it, and are now going to meet with the FDA and then hopefully file for a biologics license. Hopefully, administration chaos won’t delay consideration.