August 2026: This month in Huntington’s disease research

August has been a packed month for Huntington’s disease (HD) research. We’ve looked at what can be learned from disappointing clinical trialclinical trial Very carefully planned experiments designed to answer specific questions about how a drug affects human beings news, seen new studies launch, explored completely new ways of lowering huntingtin, and reported from one of the biggest meetings in the HD research conference circuit. Let’s get into what we learnt in HD research this month. 

Learning from setbacks: where does HTTHTT one abbreviation for the gene that causes Huntington’s disease. The same gene is also called HD and IT-15 lowering go after Roche?

We began August by looking back at Roche’s decision to discontinue two huntingtin-lowering programmes – tominersen in GENERATION HD2 and RG6496 in POINT-HD. Four weeks on from that disappointing news, we took stock of what these programmes have taught us. Both drugs successfully engaged with the biology they were designed to target, but that did not translate into the clinical benefits researchers hoped to see.

That raises some big questions for the field. Are we lowering the right forms of huntingtin? Are drugs reaching the right brain regions and cell types? How much huntingtin needs to be lowered, and when in the course of HD should treatment begin? And are the biomarkers we currently use giving us the full picture?  And how can we refine our biomarkers to better reveal what these treatments are doing in the brain? These aren’t arguments for abandoning huntingtin lowering. Instead, they show just how much more there is to learn about doing it effectively.

Huntingtin lowering takes some new routes

Other stories this month showed just how quickly researchers are expanding the HTT-lowering toolkit. The INSIGHTT trial began dosing participants with SRP-1005, an experimental RNA interferenceRNA interference A type of gene silencing treatment in which specially designed RNA molecules are used to switch off a gene therapy developed to reduce huntingtin production. 

What makes SRP-1005 stand out is its delivery: rather than requiring brain surgery or injections into the spinal fluid, it is given by an injection under the skin. That would be a major practical advantage if the approach ultimately proves safe and effective. For now, INSIGHTT is an early-stage study, so its main job is to test safety and understand how the drug behaves in people.

We also explored new routes to huntingtin-lowering therapies. Increasingly, scientists are questioning whether all forms of huntingtin should be treated as equivalent. Full-length huntingtin is only part of the picture: shorter and potentially more harmful forms, including HTT1a, may offer more precise targets.

Together with approaches that act in different tissues or use different molecular technologies, this work points towards a future where “HTTHTT one abbreviation for the gene that causes Huntington’s disease. The same gene is also called HD and IT-15 lowering” may not describe one treatment strategy, but a whole family of them.

PRECISE-HD gives pridopidine another test

HTTHTT one abbreviation for the gene that causes Huntington’s disease. The same gene is also called HD and IT-15 lowering isn’t the only therapeutic approach moving forward. The first participant has now been dosed in PRECISE-HD, a new study of pridopidine in a more specific group of people with early- to mid-stage HD.

Pridopidine has a long history in the HD field. Most recently, the Phase 3 PROOF-HD trial did not meet its primary or secondary endpointssecondary endpoints Additional questions asked in a clinical trial that help scientists look at treated patients as broadly as they can to determine the effects of a drug overall. However, analyses of participants who were not taking anti-dopaminergic medicines suggested positive signals that Prilenia believes warrant further testing.

PRECISE-HD is designed to investigate that hypothesis in a new trial. This type of subgroup analysis from an earlier study can generate an interesting idea, but a properly designed trial is needed to test whether it holds up. As always, HDBuzz will follow the data to see where this new chapter in the pridopidine story takes us.

HD2026: three days at the cutting edge of HD science

HDBuzz also reported across all three days of the HD2026 Milton Wexler Biennial Symposium, hosted by the Huntington’s Disease Foundation. The breadth of science was enormous!

Somaticsomatic relating to the body CAG expansion remained a major theme, with researchers digging deeper into the DNA repair machinery that allows CAG repeats to grow in vulnerable cells and exploring ways this process could potentially be slowed. But the meeting went far beyond DNA repair. Scientists presented advances in human genetics, artificial intelligence, biomarkers, brain circuitry and huntingtin biology. 

One particularly interesting theme was the growing effort to connect different layers of HD biology. How does expansion of the CAG repeatCAG repeat The stretch of DNA at the beginning of the HD gene, which contains the sequence CAG repeated many times, and is abnormally long in people who will develop HD alter huntingtin? Which forms of huntingtin are most damaging? Which cells are affected first? And how can those insights be transformed into treatments? Rather than searching for a single explanation for HD, the field increasingly seems to be connecting the dots between Huntingtin biology at the level of DNA, RNARNA the chemical, similar to DNA, that makes up the ‘message’ molecules that cells use as working copies of genes, when manufacturing proteins., protein, cells and ultimately symptoms in people.

Making every dataset count

Another August story showed that scientific progress doesn’t always require generating new data. Prilenia shared placebo-group data from two HD clinical trials with CHDI, making these valuable datasets available for further research. Participants assigned to take placeboplacebo A placebo is a dummy medicine containing no active ingredients. The placebo effect is a psychological effect that causes people to feel better even if they’re taking a pill that doesn’t work. contribute enormous amounts of information about how HD causes changes over time, and combining datasets can help researchers understand disease progression, placeboplacebo A placebo is a dummy medicine containing no active ingredients. The placebo effect is a psychological effect that causes people to feel better even if they’re taking a pill that doesn’t work. effects and how future trials might be designed more efficiently.

A clinical trialclinical trial Very carefully planned experiments designed to answer specific questions about how a drug affects human beings therefore doesn’t stop being useful when its original question has been answered. Sharing high-quality data means the contributions made by participants can continue generating knowledge for years to come.

Growing the next generation of HD communicators

Finally, August saw the launch of the 2026 HDBuzz Prize for Young Science Writers. HD science is moving quickly and becoming increasingly complex. Making that science understandable without losing key details is essential if the HD community is to participate in conversations about where research is heading. Supporting young researchers to develop those skills helps ensure that clear, accurate and accessible HD communication continues into the next generation.

Many paths, one goal

August really illustrated the current HD research landscape and the diverse research ideas being investigated in this field. Some researchers are figuring out what we can learn from trials that didn’t work. Others are testing new drugs, devising more precise ways of targeting huntingtin, investigating the DNA changes that occur over a lifetime, or opening up old datasets to answer new questions. Not every route will lead to an effective treatment.  But pursuing diverse ideas, and making sure we learn from every result, gives the HD community the best chance of finding those that do.

Summary: 

• In August 2026, Roche’s recent setbacks raised important questions about what, where, when, and how much huntingtin we should lower, while new research is exploring more targeted ways to tackle harmful forms of the protein.
• New clinical trials kicked off with INSIGHTT testing the HTT-lowering drug SRP-1005, while PRECISE-HD is giving pridopidine another test based on hypotheses generated by earlier clinical trialclinical trial Very carefully planned experiments designed to answer specific questions about how a drug affects human beings data.
• HD2026 showcased a rapidly expanding research landscape, spanning somaticsomatic relating to the body CAG expansion, DNA repair, HTTHTT one abbreviation for the gene that causes Huntington’s disease. The same gene is also called HD and IT-15 biology, biomarkers, brain circuitry, artificial intelligence, and new therapeutic ideas.
• August also highlighted the value of sharing clinical trialclinical trial Very carefully planned experiments designed to answer specific questions about how a drug affects human beings data and training the next generation of scientists to communicate HD research clearly.