uniQure’s AMT-130 at the forefront of 21st Huntington’s Disease Therapeutics Conference

  

AMT-130, the uniQure gene therapy that successfully slowed the progression of Huntington’s disease before being obstructed from approval by the U.S. Food and Drug Administration (FDA), stands at the forefront of the leading HD research conference that starts today, February 23, in Palm Springs, CA.

 

The 21st Annual Huntington’s Disease Therapeutics Conference, which I have described as the Super Bowl of HD research, takes place at the Parker hotel. It is sponsored by CHDI Foundation, Inc., the largest private funder of HD research.

 

“In recent years HD drug discovery has become increasingly anchored in strong human genetic and clinical evidence, allowing us to focus on mechanisms most likely to yield disease-modifying benefit,” wrote Robert Pacifici, Ph.D., the CHDI chief scientific officer, in his welcome letter to the conference attendees. “Few developments capture this momentum more clearly than the recent topline results announced by uniQure from the pivotal Phase I/II study of AMT-130 suggesting that lowering [decreasing] mutant huntingtin in people confers a real clinical benefit, an important proof of biological principle.”

 

Dr. Pacific did not refer to the FDA roadblock but asserted that the AMT-130 “findings represent an encouraging milestone for the HD community.”

 

For its clinical trial analysis uniQure has relied on the CHDI-backed Enroll-HD, the global registry of HD-affected individuals and their families. uniQure has presented its data at the therapeutics conferences.

 

Dr. Robert Pacifici, wearing a Team Hope shirt from the Huntington's Disease Society of America, overseeing the 2025 Therapeutics Conference (photo by Gene Veritas, aka Kenneth P. Serbin)

 

AMT-130 talk in the lead-off

 

Dr. Pacifici and the conference planners scheduled a report on AMT-130 as the first presentation at the start of the opening science session on February 24.

 

David Margolin, M.D., Ph.D., uniQure’s vice president for clinical development, will give a talk titled “AMT-130 slows Huntington's disease progression at 3 years: Propensity score weighting mitigates potential bias from striatal volume absence in Enroll-HD.”

 

Because AMT-130 has been administered via a spinal injection that involves 12-plus hours of surgery, uniQure had no placebo group – those not getting an operation –but instead chose to use data from Enroll-HD as a comparison group.

 

After extensive consultations with the FDA in 2024, uniQure had gotten permission from the agency to use the Enroll-HD data. In November 2025, the FDA told uniQure that Enroll-HD data might no longer be “adequate.”

 

The HD community mobilized immediately, garnering 48,000-plus signatures on two petitions delivered to FDA headquarters on January 22.

 

The abstract of Dr. Margolin’s presentation does not reference the FDA but states that the Enroll-HD data about loss of striatal volume (shrinkage in the striatum, located deep in the brain and severely affected in HD) was helpful in the analysis of the clinical trial data. He reasserted the importance of MRI measurements of such brain loss.

 

Awaiting news about the FDA

 

uniQure announced on January 9 that it had scheduled a high-priority meeting with the FDA, which according to regulations had to occur within 30 days. The FDA must produce final minutes of the meeting.

 

As of this writing, uniQure has not reported on the results of the meeting.

 

At the conference, the several hundred scientists, biopharma reps, and advocates like me will anxiously await the latest news on AMT-130.

 

Stay tuned for my reports on the event.

‘Ready to fight the fight’: Huntington’s disease community delivers petitions to FDA seeking approval of uniQure gene therapy

  

On January 22 Huntington’s disease advocacy organizations delivered two petitions to the U.S. Food and Drug Administration (FDA), demanding that the agency reverse its rollback on uniQure’s application for its HD gene therapy.

 

In September, uniQure announced that its drug, AMT-130, had slowed the progression of HD by 75 percent over three years – a historic first. However, reflecting what critics saw as growing dysfunction under the Trump administration, the FDA abruptly switched gears regarding its agreed-to plan for considering AMT-130.

 

HD advocates quickly organized two online petitions. By January 17, they had garnered more than 48,000 signatures.

 

Advocates from HD Reach, Help4HD International, the Huntington's Disease Foundation, the Huntington’s Disease Society of America (HDSA), and the Huntington’s Disease Youth Organization (HDYO) jointly delivered the printed petitions to FDA headquarters in Silver Spring, MD.

 

“We can’t delay any longer,” Lauren Holder, a Help4HD advocate and (like me) an HD gene carrier desperate for a therapy like AMT-130, said in a Facebook video. “It’s important that they hear us.”

 

Surviving HD

 

The 48,000-plus signatures “is not something that the HD community has ever done before,” Holder said. In addition, the “outcry” by advocates to Congress was highly effective, she added.

 

The HD community’s “wonderful job” in speaking up is “important for clinical trials in the future” of gene therapies and other gene-modifying drugs, she said.

 

Holder said that she often hears from HD-affected individuals that “‘I don’t want to be dying from HD; I want to be living with HD.’ These gene therapies and gene-modifying drugs are what get us to that point.”

 

Ramping up the fight

 

In a final, intense push for signatures, Holder on her January 16 podcast interviewed Jeremy Renz, of St. Augustine, FL. His wife tested positive for the HD gene in 2024 and his mother-in-law is in the late stages of the disease. Their family has been affected for many generations.

 

In 2024, Renz was a panelist at the FDA headquarters for a listening session with the HD community, similar to the first such meeting organized by HDSA in 2015, which I attended.

 

Whereas Help4HD and other organizations advocated for one petition, Renz organized another for those families not necessarily linked to formal HD groups.

 

“There’s more young people that are testing,” Renz told Holder. “There’s more young people who are at risk that are getting ramped up and ready to fight the fight.”

 

HD family member Jeremy Renz with petitions advocating for the approval of uniQure's AMT-130 at FDA headquarters, Silver Springs, MD, January 22, 2026 (Facebook photo)

 

A crucial meeting

 

On January 9, uniQure announced that it had scheduled a so-called Type A (high-priority) meeting with the FDA, to take place within 30 days, “to support accelerated approval of AMT-130.”

 

On January 20, Daniel Leonard, uniQure’s executive director of global patient advocacy, distributed a letter to the HD community explaining the purpose of the Type A meeting.

 

“Type A meetings are intended to address urgent issues and help a sponsor, such as uniQure, resolve developmental roadblocks and obtain critical feedback from the FDA,” Leonard wrote. “The extraordinary efforts of the HD community over the past two months have played an important role in raising awareness of the significant unmet need in HD.”

 

Crucially, the Type A meeting will include a representative of the HD community, as of now unnamed.

 

Leonard stated that uniQure will provide an update after receiving the official meeting minutes from the FDA.

 

The HD community has made a difference

 

In their podcast conversation, Holder and Renz noted that the FDA’s backtrack on the AMT-130 plans was “infuriating.”

 

“It’s really, really frustrating for a community that deserves a lot,” Renz said. “We deserve an answer. We deserve treatment. And the time is now.”

 

For HD gene carriers like herself, Holder said a delay in receiving a drug such as AMT-130 reduces the “window” of opportunity for treatment.

 

Holder and Renz concluded by saluting how the HD community had united to work hard on AMT-130 advocacy.

 

“We are definitely stronger together,” Holder said. “We are living history right now. This is something that everybody is going to look back on and say: ‘The community made a difference here, and they pushed through at the right point.’”

Huntington’s disease community urges FDA to get on the right side of history for uniQure gene therapy

 

Even as uniQure, in a December 4 press release, has reconfirmed – based on meeting minutes – the decision by the U.S. Food and Drug Administration (FDA) to roll back its permission to apply for a Huntington’s gene therapy drug approval, more than 43,000 people have signed two petitions demanding the agency to uphold its original plan.

 

As recounted here, uniQure and others in the frustrated biotech sector believe that the FDA has become dysfunctional under the Trump administration.

 

In September, uniQure announced that its drug, AMT-130, had slowed the progression of HD by 75 percent over three years – a historic first.

 

Then, on November 3, uniQure announced that, after its October 29 meeting with the FDA, the agency had abruptly switched gears regarding AMT-130.

 

On December 8, the life sciences hub BioSpace reported that “rare disease leaders” want “regulatory consistency” after a “chaotic year” at the FDA.

 

Overcoming the ‘whiplash of the FDA’s about-face’

 

The key biotech site STAT has continued to report on the crises in leadership and turmoil at the FDA.

 

As of December 9, two online petitions to the FDA from HD advocates have garnered more than 43,000 signatures.

 

Click here and here for the petitions.

 

In the words of the scientist-written site HDBuzz, “The HD community has not remained silent through the whiplash of the FDA’s about-face from just five months prior, when they stated that data from the ongoing trials would be sufficient to support accelerated approval.”

 

Honors for AMT-130 researcher

 

“We are committed to collaborating with the FDA to advance AMT-130 to patients and their families as rapidly as possible,” CEO Matt Kapusta stated in the company’s December 4 release. “The support we have seen these last weeks from the Huntington’s disease community, including patients, families, caregivers, clinicians and advocates, reinforces the urgency of the unmet need in Huntington’s disease.”

 

Further validation of uniQure and the AMT-130 clinical trial came on December 8, when the key journal Nature announced that Sarah Tabrizi, M.D., Ph.D., a leading HD specialist at University College London and one of the medical leaders of the AMT-130 clinical trial, is “part of Nature’s 10, a list of people who shaped science in 2025.”

 

Titled “Sarah Tabrizi, Huntington’s hero,” the article about her describes her decades-long efforts to treat HD.

 

“I want to see if we can prevent Huntington’s from ever occurring,” Dr. Tabrizi told Nature.

 

As we approach the holiday season and the hopes that HD advocates will persuade the FDA to resume its support of the agreed-to plan for AMT-130, the recognition of Dr. Tabrizi and the community’s massive efforts signal that the FDA should be on the right side of history in this urgent fight to end devastating diseases.

 

Dr. Sarah Tabrizi at the 2020 Huntington's Disease Therapeutics Conference, Palm Springs, CA (photo by Gene Veritas, aka Kenneth P. Serbin)

As uniQure seeks to overcome ‘dysfunction’ at the FDA, Huntington’s disease community rallies in defense of gene therapy drug

As uniQure seeks to overcome a decision by the U.S. Food and Drug Administration (FDA) to roll back its consultations regarding the firm’s promising gene therapy for Huntington’s disease, the HD community has begun to rally by organizing two petitions asking the agency to support the remedy.

 

uniQure and others in the biotech sector believe that the FDA has become dysfunctional under the Trump administration.

 

In September uniQure announced that its drug, AMT-130, had slowed the progression of HD by 75 percent over three years.

 

On November 3, uniQure announced that, after a recent meeting with the FDA, it believes that the “FDA currently no longer agrees” that data from its clinical trial of AMT-130 "may be adequate to provide the primary evidence in support of” an application for approval.

 

The company’s plan to seek approval of AMT-130 in early 2026 and launch it into the market later that year may no longer be possible.

 

Although the FDA claims it will “unleash gene therapies,” the documented collapse of the agency under the Trump administration, a more conservative view of gene therapies, and “mistrust and paranoia” in the division in charge of those therapies set the stage for backtracking on AMT-130 (click here to read more).

 

On November 13, the key biotech site STAT reported on a private dinner held by uniQure CEO Matt Kapusta with investors on November 11. According to the report, uniQure hopes to find a way forward for AMT-130.

“We remain fully committed to people living with HD, who have no disease-modifying treatment options,” Tom Malone, uniQure’s senior director of communications, wrote me via e-mail on November 14. “We are wholly focused on working with the FDA to determine the best path forward to rapidly bring AMT-130 to patients and their families in the U.S.”

The company is withholding further public comment until it receives official final minutes of its most recent meeting with the FDA.

Two groups of HD advocates have launched petitions to the FDA to support the original uniQure timeline for AMT-130. They are discussed below.

 

 

The online petition to the FDA titled "Bring Hope to Huntington's Disease Families," on November 15, 2025 (screenshot by Gene Veritas, aka Kenneth P. Serbin)

 

Deep frustration with the FDA

 

The STAT report noted that Kapusta “doesn’t like all the drama” inside the FDA surrounding its upending of uniQure’s plans. They had involved extensive consultations with the FDA in 2024.

 

Aiming to stabilize the agency, the FDA has named Richard Pazdur, M.D., a leading cancer specialist and 26-year veteran of the entity, to run its center for regulating and approving new drugs.

 

Dr. Pazdur’s appointment is a sign the Trump administration is seriously addressing the “FDA dysfunction,” Kapusta said at the investor dinner, as reported by STAT.

 

According to the STAT report, Kapusta’s remarks at the dinner “reflected biotech’s frustration with volatility” at the FDA. uniQure was disturbed by the fact that the FDA’s new message on AMT-130 was “delivered by lower-level staffers” and not senior decision-makers, STAT reported.

 

The firm “feels like it was screwed over by the FDA, and rightfully so,” one investor told the STAT reporter.

 

Meanwhile, the FDA and some scientists have moved ahead with researching and approving the world’s first personalized gene-editing treatments for individuals with rare genetic conditions. It is unclear how much this might benefit rare disease communities like HD (with thousands of affected individuals) and impact the FDA’s thinking on AMT-130.

 

Two petitions: ‘AMT-130 could change everything’

 

As of November 15, the two online petitions to the FDA from HD advocates have already garnered almost 9,000 signatures.

 

These moving, persuasive petitions effectively portray the devastating impact of HD on patients and families and the historic breakthrough towards a treatment achieved with AMT-130. They effectively demonstrate the profound need for the drug and urgent action by the FDA.

 

One petition is titled “Bring Hope to Huntington's Disease Families: Urge the FDA to Uphold Accelerated Approval.”

 

It is sponsored by the Huntington’s Disease Society of America, HD Reach, Help4HD International, Huntington’s Disease Foundation, and Huntington’s Disease Youth Organization. These organizations have also pledged to improve collaboration.

 

It notes that “the FDA is now wavering on its commitment” to AMT-130. It asks the agency to honor its previous guidance to uniQure, recognize the urgency of the unmet dire medical need of HD families, and to expedite the approval of AMT-130.

 

It urges people to sign: “We cannot allow procedural hesitation to become a death sentence.”

 

The other petition is titled “Accelerate Breakthrough Drug Approval for Huntington's Disease - UniQure AMT-130.”

 

“Our Plea: Turn Heartbreak Into Hope,” it states. “We are mothers and fathers, sons and daughters, brothers and sisters, husbands and wives, friends and caregivers – all united by love and by loss. AMT-130 could change everything.”

 

As an HD gene carrier and HD family member, I immediately signed the first petition upon learning about it. I urge all friends and supporters of the HD community to join us in our plea to the FDA.