Day 3 of the HD Therapeutics Conference is bringing even more breakthrough science, new tools, and bold ideas pushing Huntington’s disease research closer to better treatments. Read today's article for the latest updates from the conference.
Full Article: en.hdbuzz.net/huntingtons-...
These sessions highlighted techniques that allow scientists to zoom in on Huntington’s disease biology in extraordinary detail, helping researchers better understand what’s happening inside cells and how it might be targeted with future treatments.
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HDBuzz is back with Day 2 of the 21st annual CHDI HD Therapeutics Conference in Palm Springs.
HDBuzz was at the HD Therapeutics Conference, and brought you live updates on the latest research in Huntington’s disease.
Read up on the exciting science, new discoveries, and insights straight from the researchers working to move the field forward.
en.hdbuzz.net/huntingtons-...
From scientists in the lab to the research participants driving discoveries forward, we recognize the women shaping the future of science and medicine.
This International Women’s Day, we celebrate the dedication, courage, and brilliance of all the women moving research forward every single day.
An oral Huntington’s disease drug has been deemed eligible for accelerated approval in Australia. While this isn’t full approval just yet, it creates a faster regulatory pathway that could help bring this once-daily pill to people living with HD sooner.
en.hdbuzz.net/australia-op...
The FDA wants more data before approving AMT-130 for Huntington’s disease in the U.S. On March 2026, uniQure shared in an update that current Phase 1/2 data weren’t enough for the agency. A new randomized, sham-controlled trial may be required en.hdbuzz.net/the-road-ahe...
Vico Therapeutics’ Huntington’s disease drug VO659 is now being tested with dosing just twice a year, and the FDA has cleared the path for US clinical trials to begin later this year.
Full article: en.hdbuzz.net/vicos-trial-...
On Rare Disease Day, we honour the patients, caregivers, clinicians, and researchers who push for answers, better treatments, and equity in care. Awareness sparks research. Research sparks hope.
Today we stand with the millions of individuals and families around the world living with a rare disease. Though each condition may be uncommon, together this community is powerful, resilient, and impossible to ignore.
And that wraps the #HDTC2026 meeting! As always, we’re floored by the novelty and diversity of ideas moving us closer to treatments for HD. Summary articles will follow, so stay tuned!
Claudia has applied a bottom-up approach to connect very rare symptomatic observations with new genetic causes. She is also applying meta-analyses that combine findings from multiple studies for an even richer collection of “big data.” #HDTC2026
To overcome this challenge, there are ongoing efforts to diversify these databases with participation from folks who come from all over the world. Hopefully this means that moving forward, gene-health connection research can be more inclusive. #HDTC2026
This kind of analysis is only made possible because of the tens of thousands of folks who participate in data collection efforts. However, these databases are dominated by people of European ancestry, which means these trends might not hold true for people from other backgrounds. #HDTC2026
Claudia and her powerhouse team have applied their analysis to see how well they are able to predict different diseases based on data signatures alone. They have had success with multiple myeloma, a type of blood cancer, and are expanding their approach to other diseases, including HD. #HDTC2026
Instead of considering proteins in isolation, they are looking at many readouts - genetics, proteomics, and other data, all in combination. This is called a multi-omics approach. Using AI tools, they can pull out patterns too complicated to spot with regular statistical approaches. #HDTC2026
Claudia shows us a huge dataset where most diseases do not have signals in their protein signatures that track robustly with disease - this is disappointing, but there are other datasets to consider. #HDTC2026
One of the biggest and best databases to study the gene-health connection is the UK Biobank. Big pharmaceutical companies and academic scientists have been analysing data to try and find links. The problem is that biology is blooming complex so the trends are not easily found. #HDTC2026
Science for the last ~10 years has been in its “big data” phase, which science calls “omics”. We have tons of data which clever scientists like Claudia are trawling through with cool computing tools to look for trends and patterns to better understand diseases, and maybe develop new drugs. #HDTC2026
Claudia reinforces the fact that new tools are allowing us to connect genetics with human health measures and symptoms on a large scale and like never before. This can help us diagnose diseases and predict their course. #HDTC2026
The next talk is from Claudia Landenberg, who has positions in both London and Berlin. Her work uses large-scale data on both the molecular and clinical level to understand the effects of genetics on cells and organs in different diseases. #HDTC2026
The next talk is from Claudia Landenberg, who has positions in both London and Berlin. Her work uses large-scale data on both the molecular and clinical level to understand the effects of genetics on cells and organs in different diseases. #HDTC2026
They also worked in partnership with Veronica Brito’s lab, who gave a talk earlier this conference. This system worked in her lab’s models too - they could see repair in some blood cells. This suggests that blood samples from people with HD could be used to track somatic expansion. #HDTC2026
This is an interesting new way to measure DNA damage repair. One takeaway is that there are many more successful repair events than events where repair goes awry to lengthen CAG repeats. Good to know that biology works most of the time! #HDTC2026
The HTT gene repair signal was impacted by blocking these genes known to impact HD, but the other repair hot spots were not affected. #HDTC2026
Next they looked to see how this EdU signal might change if different DNA repair machinery components were blocked, like MSH2, MSH3, MSH6 and PMS1 (all known to affect age of HD symptom onset. #HDTC2026
They looked to see where else EdU was added into the genome during repair, finding good signal in repair hot spots and suggesting the new approach was working well. Paolo's team are now confident they can see where repair is occurring, and therefore infer where expansion has happened. #HDTC2026
This experiment was a success! They could see the EdU but only in the HTT gene of cells which model HD, and not in healthy cells. The signal became more obvious in the HD cell models with longer and longer CAG numbers. #HDTC2026
In a proof-of-concept experiment, the scientists feed a special type of DNA letter to the neurons, called EdU (pronounced E-D-U), which they can measure as it is used to fill in these gaps in the expansion bubbles in the HTT gene. #HDTC2026
Mismatches, the bubble-outs in the DNA that often occur with expanded CAGs, are repaired with new DNA letters to fill in the gaps they create. Paolo suggests that we can infer somatic expansion by looking out for this newly synthesised DNA in the cell. #HDTC2026
