Additional Clarity: What We Know 4 Weeks After the uniQure News

The past 4 weeks have been a whirlwind in the Huntington’s disease (HD) community. On September 25th we had an update from uniQure about a drug they’re testing for HD in ongoing clinical trials. The news was positive and it took the world by storm, producing jaw dropping headlines from news sources around the world, generating global interest in HD, and prompting many people within the HD community to reach out to neurologists and care centers around the world with various questions. 

Now that the dust has settled, we can take a step back to break down what we know, where the uncertainties lie, and address and answer some of the questions we’ve heard from the HD community. Given past disappointments, it’s natural for the HD community to approach new developments carefully. We share this caution, as well as optimism, and want to frame the buzz that the recent news has generated through a realistic lens. 

uniQure’s Announcement

Just a few weeks ago, uniQure announced positive topline results from their Phase 1/2 trial of AMT-130, a gene therapy being tested in people with HD. The therapy, delivered directly into the brain through a ~10 hour surgery, is designed to permanently lower production of the HTT protein that causes HD. 

According to uniQure’s update, trial participants receiving the high dose of AMT-130 who have been followed for 3 years have experienced a 75% slowing in overall disease progression as measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS). The cUHDRS is a collection of tests that assess how well people with HD are functioning day-to-day through memory, movement, and mood tests. The improvement on the cUHDRS scale meant that the trial met its primary endpoint. 

This positive news represents a milestone, showing for the first time that a drug can alter the course of HD in people. uniQure shared that their next steps are to meet with the U.S. drug regulator, the FDA, later this year, aiming to file for accelerated approval in early 2026.

However, we’re not across the finish line yet and don’t yet have a bona fide HD treatment in hand. This is a Phase 1/2 trial, so it’s only being tested in a small group of people. Even with these critical caveats that should elicit some caution, many media outlets headlined the exciting part of the news without doing much to temper excitement. 

Given past disappointments, it’s natural for the HD community to approach new developments carefully. We share this caution, as well as optimism, and want to frame the buzz that the recent news has generated through a realistic lens.

Overhyped & Misleading Media Coverage

Unfortunately, many news outlets are driven by attention. They want your eyes on their website. So the more clicks they get the better. And big, over-the-top headlines generate a lot of clicks. Thus, when fantastic research news about a devastating brain disorder, like HD, comes out for the first time, the splashier headlines are a common tactic for some news outlets. But this can be detrimental to the very populations that the news is intended to serve. Let’s get into some of the flagrant headlines that made our eyes pop and jaws drop.

HD “Successfully Treated”?

Some of the more egregious headlines claimed that HD was “successfully treated” for the first time, even pairing this news with pictures of world-renowned, incredibly reputable HD researchers. Because of that, it’s understandable that anyone with HD, from an HD family, or who even knows anyone affiliated with HD would immediately get on the phone to call or text about the next steps. 

First and foremost – was HD successfully treated for the first time? The jury is still out on this one; the data are promising, but not conclusive. The recent news from uniQure does not yet show that we have a successful treatment for HD. It shows in a small number of people being given AMT-130 that metrics measuring signs and symptoms of HD are moving in a favorable direction that suggests the drug may be able to slow disease progression. 

Is this good news? Undoubtedly. Is it conclusive? By no means. AMT-130 is currently being tested in a Phase 1/2 trial. Even if this trial is wildly successful and surpasses every expectation, we will still need more data to conclusively say that it is a successful drug in treating HD. 

uniQure has always been transparent about the fact that even if this trial is successful, the next steps could include accelerated regulatory approval, which is different from a fully approved drug on the traditional path. Drugs that receive accelerated approval can be marketed with the understanding that more data will be collected in larger trials with more trial participants to ensure that the drug can do what everyone hopes it will do. If it doesn’t, the accelerated approval label will be revoked, the drug will be pulled from market, and it will no longer be sold. Fully approved drugs have been tested in a sufficient number of people to conclusively show that they work as intended, and they likely won’t be pulled from market for efficacy reasons. 

A “Breakthrough Cure”?

Other conclusive-sounding headlines suggested that the world had found a “breakthrough cure” for HD. Similar to headlines claiming we’ve “successfully treated” HD, the data do not yet support the conclusion that we have a cure for HD. 

A cure implies that someone who once had HD no longer has the disease, which suggests that there has been a reversal of symptoms or elimination of all signs of HD. The data from uniQure do not suggest that HD has been cured or that HD signs and symptoms are being reversed. The data suggest that progression of HD is being slowed. While this is still fantastic news, it’s an incredibly critical difference.

Some Weren’t So Bad

Not every news source put out a headline that exaggerated the results. Some of the more level-headed articles underscored that the results “show promise”, were “a cause for hope”, were “preliminary but promising”, and “slowed progression”. 

Science is rarely cut and dry, especially when it’s in earlier stages like a Phase 1/2 trial, so headlines that are more cautious and avoid conclusive sounding language are usually more in line with a level interpretation of the findings. 

Navigating Headlines 

A sure fire way to spot articles that you should view with some skepticism are those with huge claims, like a successful treatment or cure for HD. At HDBuzz, we recently updated an article with 10 golden rules you can use to navigate HD research news. With all the news swirling about, if you haven’t checked this article out, now would be a fantastic time to give it a glance so that you’re brushed up on your HD media literacy. 

Some of the more level-headed articles underscored that the results “show promise”, were “a cause for hope”, were “preliminary but promising”, and “slowed progression”.

The Key Caveats

We mentioned that there are some key caveats that should elicit caution around these results. So, what are they?

Small number of participants

As stated above, this is an early, small trial. Phase 1/2 trials by design are intended to be small because we don’t want to test unproven drugs in large numbers of people without more evidence that they may be successful. Trials are designed to include increasing numbers of participants as evidence mounts that the drug is likely to work. 

Because of that, this ongoing trial has just 29 participants – 12 in the low dose group, and 17 in the high dose group. Of those 17 receiving the high dose, 12 have made it to the 3 year mark, so the exciting data currently being scrutinized is from just those 12 people. This isn’t a very big group. 

Trials with larger numbers of people evoke more confidence because it’s less likely that a larger group of people would all have some sort of biological anomaly chosen by chance. For example, it could be possible that 12 randomly selected people may all have some factor (biological, pharmalogical, or otherwise) that may make them perform better on a certain drug, but this chance decreases if you have 100 people, for example. Inclusion and exclusion criteria try to account for some of this, but smaller sample sizes still have this probabilistic risk. 

The comparator group

Very robust clinical trials are compared against a placebo group – a group of people that are similar to the participants being given the drug that are instead given a sugar pill, or a mock surgery, as it would be in this case. While the first 12 months of this study were tested against such a group, after that initial year, those people were given the option of receiving the drug. Unfortunately because this study, and any gene therapy study, is long term, some of those people were no longer eligible for the drug based on the inclusion and exclusion criteria for the trial. 

Thus, moving forward the trial was controlled against data from Enroll-HD, which is an observational study that follows people with HD as they naturally live and age. For this reason, the comparator group in this trial is much larger, following over 1500 people, 940 of whom are being compared against the high dose group. 

This is a less rigorous way to test a drug for several reasons. Firstly, there’s no easy way to determine that “the placebo effect” isn’t improving their symptoms. The mind is incredibly powerful over the body! Ample research shows us that the suggestive powers of just thinking you’re taking something that might help you get better and can actually reduce symptoms. Secondly, people who are frequently seen and looked after by doctors, nurses, social workers, and therapists, like participants of a clinical trial, often have improved symptoms, regardless of what drugs they are receiving. The people in this trial could be going to the clinic more frequently than those in the Enroll-HD comparator group, causing a medical response bias that improves their symptoms. 

However, while comparator groups are a less rigorous way to test a drug, they’re often considered a more ethical way to test a gene therapy drug in early stages. As drugs advance through the regulatory process and reach later clinical phases, double-blinded, placebo-controlled trials could be required. So for a Phase 1/2 trial like this one for AMT-130, a natural history comparator can give us an idea if a drug is meeting certain metrics, but isn’t robust enough to conclusively tell us if a gene therapy is effective. 

Other considerations 

There are also a variety of other considerations that researchers are pondering, but don’t yet have the data to support or produce conclusive results around. 

Are there people who will respond well to this treatment while others won’t? Given the small number of people in this trial, it’s possible they could be “super responders” – people who respond particularly well to a specific treatment. If so, what might be the delineating factors that define these subgroups? Is it developmental brain stage, disease stage, age, etc? We’ve not seen data from individuals treated with AMT-130 yet, only the group averaged together. Once we have this data, we could get some clarity around these questions.

Even with the permanence of this treatment, how durable will it be? Will it wear off over time? Might additional treatments be needed in the future? If so, how far out and at what dose? This is complicated because we don’t have satisfactory tools or surrogates to accurately measure HTT lowering, particularly in brain tissue. 

We’ve not yet seen all the data collected from this trial and it’s not yet been peer-reviewed or published. How will these data measure up when examined by other researchers? 

With any gene therapy, accessibility will be an issue. Would this treatment be covered by insurance companies? If not, how will HD families afford such treatments that can run into the millions of dollars?

In all, there are still many outstanding questions to consider for which we just don’t yet have answers. 

Treatment-associated risks

Even if all the other considerations prove favorable, there are still two large potential treatment-associated risks that should be considered around AMT-130. This is a permanent gene therapy that is delivered via brain surgery. 

Firstly, while there are very skilled brain surgeons carrying out these surgeries, brain surgery is never without risk. This is a highly invasive procedure. We understand that the risks around brain surgery are something that many in the HD community are willing to tolerate for a potential treatment against this disease, however it still must be stated that brain surgery always comes with a risk. 

Secondly, this is a permanent treatment. Once AMT-130 is delivered to the brain, there is no going back, good or bad. While a one time treatment could be viewed as desirable by many within the HD community, this type of non-reversible change for an unproven treatment also carries risks that must be acknowledged. 

It is for these reasons that gene therapy trials, like uniQure’s AMT-130, are rolled out slowly and carefully in a very small number of people in a staggered way. Initially, the surgery was given to only 1 person, who was followed for several months before a second person was inducted into the trial. This specific trial design was devised to mitigate safety risks around brain surgery and permanence of this treatment approach. 

So far, the data suggests there is a slowing of disease in people on the high dose of AMT-130, but the disease in those people is still progressing.

Reaction From The Community 

With the exciting results from this trial and the abundance of hyped up headlines, the HD community has understandably had a lot of questions. We’d like to address some of the more frequently asked questions we’ve heard.

Does this mean there’s finally a cure for HD?

No. But this is an important step forward. 

A “cure” implies that someone who had signs and symptoms of HD no longer does. That would require reversal of disease worsening, which is not what we’ve seen. So far, the data suggests there is a slowing of disease in people on the high dose of AMT-130, but the disease in those people is still progressing.

So, has HD been successfully treated for the first time?

We don’t know yet. But this is some of the most encouraging data we’ve seen from a clinical trial thus far. While we need data from a larger group of people to have conclusive evidence that a drug has treated HD, the recent data from uniQure is the first to show that any drug is slowing disease progression, which is incredibly encouraging.

Where can I get access to AMT-130?

AMT-130 is not yet an approved treatment, so it’s not readily available at trial sites for the general public, hospitals, or doctors offices. 

AMT-130 is still being actively tested in clinical trials. If you are interested in participating in a trial for AMT-130, you can check if the trial is enrolling at a site near you using HDSA’s HD Trial Finder and speak to your neurologist and medical care team.  

When will AMT-130 be approved?

uniQure has stated that they plan to speak to the FDA about accelerated approval for AMT-130 in early 2026. While uniQure and the FDA have had alignment on moving AMT-130 forward thus far, the path forward is not guaranteed. The FDA will determine if the data are strong enough for accelerated approval.

If the data continue to look promising and the FDA grants accelerated approval, AMT-130 could potentially be approved by the end of 2026. However it would still take time to roll this out to the general public. Additionally, if more data are required prior to approval, this timeline could be extended. 

How much will AMT-130 cost?

uniQure has not yet released the price point for AMT-130, but has stated that it will be in line with other gene therapies. Given the nature of gene therapies, they are incredibly expensive, ranging from about $2 to $4.25 million dollars for the drug alone. Depending on your country’s healthcare system, there could also be additional medical care costs to consider associated with hospital stays, anaesthesia, and brain surgery. 

It is not yet clear what costs may be covered by different national healthcare providers, insurance companies, or other health programs around the world. However, uniQure has stated that they plan to discuss the value of the drug with the different agencies who may cover costs, which could improve access if AMT-130 is approved by regulators. 

Who will be eligible to receive AMT-130 if approved?

Currently, AMT-130 is being tested in people with fairly early symptoms of HD. If the drug is approved by regulatory agencies, people who have a similar disease stage to those tested in the trial would likely be eligible initially. The goal for any drug that could successfully treat one subset of people with HD would be to broaden who may successfully be eligible, so uniQure is very likely to do subsequent studies to test the limits on who AMT-130 may successfully treat – moving both earlier and later in disease stage. 

What if I’m not eligible for AMT-130?

While we understand that not being eligible for AMT-130 could elicit strong emotions for many, there are many other treatments being developed and tested right now in a whole suite of clinical trials. The goal of every researcher working on HD is to bring a treatment for this disease forward to help as many people as possible. 

If I’m at risk, should I get tested for HD?

Getting tested for HD is a deeply personal decision. If the recent news from uniQure has made you wonder if you should get tested, we encourage you to reach out to a genetic counsellor who can help you weigh your options, as there are emotional, legal, and financial implications to consider. 

If you are based in the US, there are also considerations around eligibility for various types of insurance, including life, disability, and long-term care. 

If you are considering getting tested, you can reach out to HD Genetics, a company specializing in genetic testing for individuals at risk for Huntington’s disease, to learn more.

What can I do right now?

If the recent news from uniQure has compelled you to get involved in HD research, a great starting point can be to participate in Enroll-HD. It’s the largest observational trial (meaning no drug is given) that follows people with and without HD to collect data as they naturally live and age. 

Researchers from around the globe use the Enroll-HD database to ask and answer various questions that are consistently teaching us more about HD. Your involvement in Enroll, and any trial, helps researchers understand HD better to improve clinical trial design and advance us toward disease modifying treatments more quickly. 

Getting tested for HD is a deeply personal decision. If the recent news from uniQure has made you wonder if you should get tested, we encourage you to reach out to a genetic counsellor who can help you weigh your options, as there are emotional, legal, and financial implications to consider. 

Science Advances In Steps, Not Leaps

We know that science is incremental and often moves more slowly than anyone hopes. But it does advance. And we’ve met many milestones that have gotten us to this point. One of those is undoubtedly the September data update from uniQure about AMT-130. 

However, while we celebrate this progress, let’s do so under a realistic lens – we don’t have a treatment in hand, yet. We have to continue reading popular press articles with a critical eye, being wary of headlines that promise we’ve crossed the finish line. Getting updates about HD research from reputable sources is more important now than ever before as we near disease modifying drugs. Communicating clearly to the HD community about what results show, what it means, and what we still need to learn is critical as we move forward.

With that in mind though, please remember that HD research is moving faster than ever. Each step we’ve taken – from the description of the disease in 1872, to the discovery of the gene in 1993, to now having evidence of what we need to do to slow HD in 2025 – is taking us to the top of the mountain. It hasn’t been great leaps that have gotten us here, but rather steady progress toward a goal and a commitment from the entire HD community. With that momentum behind us, we’re nearing the final stretch. It will still take time, teamwork, and persistence, but the path is clearer than it’s ever been, and every step forward brings us one step closer to treatments that change lives.

Summary

• uniQure announced encouraging results from its Phase 1/2 trial of AMT-130, a gene therapy designed to lower huntingtin in people with HD.
• High-dose participants followed for 3 years showed a ~75% slowing in disease progression on the cUHDRS, meeting the trial’s primary endpoint.
• This marks the first time any therapy has shown the potential to alter HD progression in people, a historic milestone for the field.
• However, the study is small (29 participants, 12 in the 3-year high-dose group), and results have not yet been peer-reviewed or published.
• Some media headlines overstated the findings, with claims of a “cure” or “successful treatment.” The data show slowing of symptoms, not reversal or elimination of disease.
• The trial used a comparator group from Enroll-HD instead of a full placebo control after year one, so results should be interpreted cautiously.
• AMT-130 delivery requires brain surgery and involves permanent changes, which carry inherent risks.
• Key questions remain: durability of benefit, variability of response, affordability, and future accessibility.
• uniQure plans to seek accelerated FDA approval in 2026, but this would still require confirmatory studies and regulatory review.
• AMT-130 is not yet approved or available outside clinical trials. Interested participants should discuss enrollment options with their neurologist or consult the HDSA trial finder.
• Progress is real but incremental. This isn’t a leap forward to a cure, but a meaningful step forward on the long road to disease-modifying treatments.