UniQure's gene therapy for Huntington’s disease continued to slow disease progression four years after its implantation into patients' brains, the company reported.
However, the magnitude of the treatment’s benefit waned compared to a similar analysis conducted one year ago. https://bit.ly/3VH5Njv
However, the magnitude of the treatment’s benefit waned compared to a similar analysis conducted one year ago. https://bit.ly/3VH5Njv
UniQure's gene therapy continues to slow Huntington's progression after four years
UniQure's gene therapy continues to slow Huntington's progression after four years. But the magnitude of the benefit waned compared to a similar analysis from a year ago.
bit.ly
September 29, 2026 at 3:49 PM
UniQure's gene therapy for Huntington’s disease continued to slow disease progression four years after its implantation into patients' brains, the company reported.
However, the magnitude of the treatment’s benefit waned compared to a similar analysis conducted one year ago. https://bit.ly/3VH5Njv
However, the magnitude of the treatment’s benefit waned compared to a similar analysis conducted one year ago. https://bit.ly/3VH5Njv
Next sentence from that article:
“On Friday, Representative Jake Auchincloss, a Democrat of Massachusetts, said on social media that Dr. Prasad was the official and that he had disclosed trade secret information about UniQure’s treatment without legal authorization.”
How else would you report it?
“On Friday, Representative Jake Auchincloss, a Democrat of Massachusetts, said on social media that Dr. Prasad was the official and that he had disclosed trade secret information about UniQure’s treatment without legal authorization.”
How else would you report it?
March 7, 2026 at 2:13 AM
Next sentence from that article:
“On Friday, Representative Jake Auchincloss, a Democrat of Massachusetts, said on social media that Dr. Prasad was the official and that he had disclosed trade secret information about UniQure’s treatment without legal authorization.”
How else would you report it?
“On Friday, Representative Jake Auchincloss, a Democrat of Massachusetts, said on social media that Dr. Prasad was the official and that he had disclosed trade secret information about UniQure’s treatment without legal authorization.”
How else would you report it?
March 7, 2026 at 12:06 AM
Vinay Prasad spiking promising Huntington’s treatment without giving a reason I can see.
www.statnews.com/2025/11/03/h...
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 3, 2025 at 3:44 PM
Vinay Prasad spiking promising Huntington’s treatment without giving a reason I can see.
www.statnews.com/2025/11/03/h...
www.statnews.com/2025/11/03/h...
UniQure's gene therapy continues to slow Huntington's progression after four years. But the magnitude of the benefit waned compared to a similar analysis from a year ago.
UniQure's gene therapy continues to slow Huntington's progression after four years
www.statnews.com
September 29, 2026 at 1:32 PM
UniQure's gene therapy continues to slow Huntington's progression after four years. But the magnitude of the benefit waned compared to a similar analysis from a year ago.
Hooray! And don't ask about Uniqure's fudged preclinical data. Who cares!
forbetterscience.com/2025/09/26/s...
forbetterscience.com/2025/09/26/s...
Undoing everything Makary and Prasad did at the FDA continues.
Following dispute with FDA, UniQure is cleared to submit Huntington’s treatment for approval
The FDA has reversed its opposition to a closely watched experimental treatment for Huntington’s disease, clearing a path for its maker.
www.statnews.com
June 17, 2026 at 2:10 PM
Hooray! And don't ask about Uniqure's fudged preclinical data. Who cares!
forbetterscience.com/2025/09/26/s...
forbetterscience.com/2025/09/26/s...
Totally correct.
And if it's a single dose that cures the disease and not a lifetime course? Not a problem for capitalism! www.fiercepharma.com/pharma/csl-a...
And if it's a single dose that cures the disease and not a lifetime course? Not a problem for capitalism! www.fiercepharma.com/pharma/csl-a...
Sporting a $3.5M price tag, CSL and uniQure's hemophilia B gene therapy crosses FDA finish line
Move over, bluebird bio: There's a new most expensive drug in the world. | CSL Behring and uniQure's newly approved hemophilia B gene therapy, Hemgenix, comes with an eye-popping price tag of $3.5 mil...
www.fiercepharma.com
February 18, 2026 at 6:48 AM
Totally correct.
And if it's a single dose that cures the disease and not a lifetime course? Not a problem for capitalism! www.fiercepharma.com/pharma/csl-a...
And if it's a single dose that cures the disease and not a lifetime course? Not a problem for capitalism! www.fiercepharma.com/pharma/csl-a...
November 5, 2025 at 10:55 AM
#uniQure's #genetherapy for #Huntington's continued to slow disease progression over four years of follow-up in a clinical trial, but signs that #efficacy may be starting to wane spooked #investors.
pharmaphorum.com/news/uniqure...
pharmaphorum.com/news/uniqure...
uniQure falls on long-term Huntington's gene therapy data
uniQure's latest data drop for its gene therapy for Huntington's disease, under review in the US and UK, has spooked investors.
pharmaphorum.com
September 30, 2026 at 8:39 AM
#uniQure's #genetherapy for #Huntington's continued to slow disease progression over four years of follow-up in a clinical trial, but signs that #efficacy may be starting to wane spooked #investors.
pharmaphorum.com/news/uniqure...
pharmaphorum.com/news/uniqure...
I had my Human Genomics students analyze promising Huntington Disease trials two weeks ago and we specifically talked about uniQure's AMT-130 treatment!
September 25, 2025 at 12:09 AM
I had my Human Genomics students analyze promising Huntington Disease trials two weeks ago and we specifically talked about uniQure's AMT-130 treatment!
Possibly of some relevance, uniQure's preclinical studies on Huntington's contain fake data.
forbetterscience.com/2025/09/26/s...
forbetterscience.com/2025/09/26/s...
After yrs of disappointment scientists have made a genuine advance in treating Huntington's. The new gene therapy slowed disease progression by 75%. But it's still experimental and will likely be $$, @parshallison.bsky.social reports for @sciam.bsky.social: www.scientificamerican.com/article/firs...
How Scientists Finally Found a Treatment that Slows Huntington’s Disease
After years of heartbreak, researchers have found an experimental treatment that can slow the progression of Huntington’s disease, according to early results from a small clinical trial
www.scientificamerican.com
October 10, 2025 at 2:25 PM
Possibly of some relevance, uniQure's preclinical studies on Huntington's contain fake data.
forbetterscience.com/2025/09/26/s...
forbetterscience.com/2025/09/26/s...
The FDA has reversed its opposition to UniQure's closely watched experimental treatment for Huntington’s disease, clearing a path for the company to file for U.S. approval.
UniQure will submit a marketing application in Q3 seeking accelerated approval.
UniQure will submit a marketing application in Q3 seeking accelerated approval.
Following dispute with FDA, UniQure is cleared to submit Huntington’s treatment for approval
The FDA has reversed its opposition to a closely watched experimental treatment for Huntington’s disease, clearing a path for its maker.
www.statnews.com
June 17, 2026 at 3:00 PM
The FDA has reversed its opposition to UniQure's closely watched experimental treatment for Huntington’s disease, clearing a path for the company to file for U.S. approval.
UniQure will submit a marketing application in Q3 seeking accelerated approval.
UniQure will submit a marketing application in Q3 seeking accelerated approval.
Dr. Vinay Prasad is a jerk off.
How bad of a jerk off?
Take a read at this:
www.fiercebiotech.com/biotech/uniq...
How bad of a jerk off?
Take a read at this:
www.fiercebiotech.com/biotech/uniq...
November 18, 2025 at 2:13 AM
Dr. Vinay Prasad is a jerk off.
How bad of a jerk off?
Take a read at this:
www.fiercebiotech.com/biotech/uniq...
How bad of a jerk off?
Take a read at this:
www.fiercebiotech.com/biotech/uniq...
NEW from @adamfeuerstein.bsky.social: FDA is ‘not convinced’ UniQure’s Huntington’s therapy has benefit, senior official says
www.statnews.com/2026/03/03/u...
www.statnews.com/2026/03/03/u...
FDA is ‘not convinced’ UniQure’s Huntington’s therapy has benefit, senior official says
The FDA believes that an experimental therapy for Huntington’s disease from UniQure wasn't providing benefit for patients, a senior official told STAT.
www.statnews.com
March 3, 2026 at 1:35 PM
NEW from @adamfeuerstein.bsky.social: FDA is ‘not convinced’ UniQure’s Huntington’s therapy has benefit, senior official says
www.statnews.com/2026/03/03/u...
www.statnews.com/2026/03/03/u...
"UniQure has said it wouldn’t be feasible to run a true randomized, double-blind placebo-controlled study, considered the gold standard, because it wouldn’t be ethical to make people undergo a sham hours-long brain surgery." www.cnbc.com/2026/03/05/f...
FDA official calls UniQure's gene therapy a 'failed' treatment for Huntington's disease
UniQure needs to run another trial to prove its gene therapy "actually helps people with Huntington's disease," said a senior FDA official.
www.cnbc.com
March 5, 2026 at 10:52 PM
"UniQure has said it wouldn’t be feasible to run a true randomized, double-blind placebo-controlled study, considered the gold standard, because it wouldn’t be ethical to make people undergo a sham hours-long brain surgery." www.cnbc.com/2026/03/05/f...
Interesting podcast from the investors who helped take down FDA leaders and the influence of former Enron think tank, Arnold Ventures on the FDA.
Peter Mantas on the Coiled Spring of Biotech: FDA Shakeups, uniQure's Breakthrough, and AI Drug Discovery
Podcast Episode · This Week in Intelligent Investing · May 13 · 54m
podcasts.apple.com
May 25, 2026 at 9:16 PM
Interesting podcast from the investors who helped take down FDA leaders and the influence of former Enron think tank, Arnold Ventures on the FDA.
UniQure’s Huntington’s therapy fails to significantly slow disease progression, shares plunge
UniQure said its gene therapy for Huntington's disease fell short of statistical significance on the main goal of slowing disease progression in a four-year analysis, sending its U.S.-listed shares…
UniQure said its gene therapy for Huntington's disease fell short of statistical significance on the main goal of slowing disease progression in a four-year analysis, sending its U.S.-listed shares…
UniQure’s Huntington’s therapy fails to significantly slow disease progression, shares plunge
UniQure said its gene therapy for Huntington's disease fell short of statistical significance on the main goal of slowing disease progression in a four-year analysis, sending its U.S.-listed shares…
news-area.com
September 30, 2026 at 12:00 AM
UniQure’s Huntington’s therapy fails to significantly slow disease progression, shares plunge
UniQure said its gene therapy for Huntington's disease fell short of statistical significance on the main goal of slowing disease progression in a four-year analysis, sending its U.S.-listed shares…
UniQure said its gene therapy for Huntington's disease fell short of statistical significance on the main goal of slowing disease progression in a four-year analysis, sending its U.S.-listed shares…
UniQure's brain disorder drug slows disease progression in trial reut.rs/3IsdNic
UniQure's brain disorder drug slows disease progression in trial
UniQure's experimental gene therapy significantly slowed the progression of a brain disorder in an early-to-mid stage study, it said on Wednesday.
reut.rs
September 24, 2025 at 12:27 PM
UniQure's brain disorder drug slows disease progression in trial reut.rs/3IsdNic
FDA rejected it though for now (not formally, but they signalled they are unwilling to approve it for now)
www.biopharmadive.com/news/uniqure...
www.biopharmadive.com/news/uniqure...
January 4, 2026 at 9:41 AM
FDA rejected it though for now (not formally, but they signalled they are unwilling to approve it for now)
www.biopharmadive.com/news/uniqure...
www.biopharmadive.com/news/uniqure...
cf Uniqure’s Huntington’s disease read-out, Trifakta and Yescarta being casual standards of care, voxelotor kicking off a reverse genetics wave (ISG15, DEC2, and SCN9A are the ones I’m watching), sustained Q > 0.5, cheap robot and compute parts, PVC-eating bacteria, cultured bacon, etc
September 27, 2025 at 3:22 AM
cf Uniqure’s Huntington’s disease read-out, Trifakta and Yescarta being casual standards of care, voxelotor kicking off a reverse genetics wave (ISG15, DEC2, and SCN9A are the ones I’m watching), sustained Q > 0.5, cheap robot and compute parts, PVC-eating bacteria, cultured bacon, etc
#Huntingtondisease first treatment effectively slows disease progression by 75% now faces FDA and approval hurdles. #AMT-130
Gene Therapy Slows Huntington's Disease by 75% in Clinical Trial - Karmactive
uniQure's AMT-130 gene therapy shows unprecedented 75% slowing of Huntington's disease progression. Patients maintain abilities longer, with one returning to work. FDA approval application expected in...
www.karmactive.com
December 29, 2025 at 12:03 AM
#Huntingtondisease first treatment effectively slows disease progression by 75% now faces FDA and approval hurdles. #AMT-130
*Statement from EHDN Chairs on uniQure’s Topline Results*
EHDN Chairs Patrick Weydt (University of Bonn) and Åsa Petersén (Lund University) share the excitement of the HD community in welcoming the topline results from uniQure’s phase 1/2 study of AMT-130:
ehdn.org/statement-fr...
#Huntingtons
EHDN Chairs Patrick Weydt (University of Bonn) and Åsa Petersén (Lund University) share the excitement of the HD community in welcoming the topline results from uniQure’s phase 1/2 study of AMT-130:
ehdn.org/statement-fr...
#Huntingtons
Statement from EHDN Chairs on uniQure’s Topline Results – European Huntington's Disease Network
ehdn.org
September 30, 2025 at 9:33 AM
*Statement from EHDN Chairs on uniQure’s Topline Results*
EHDN Chairs Patrick Weydt (University of Bonn) and Åsa Petersén (Lund University) share the excitement of the HD community in welcoming the topline results from uniQure’s phase 1/2 study of AMT-130:
ehdn.org/statement-fr...
#Huntingtons
EHDN Chairs Patrick Weydt (University of Bonn) and Åsa Petersén (Lund University) share the excitement of the HD community in welcoming the topline results from uniQure’s phase 1/2 study of AMT-130:
ehdn.org/statement-fr...
#Huntingtons