Research news

Over the past year, HDBuzz has been evolving, growing, and delivering on its mission—bringing clear, accessible, and accurate Huntington’s disease (HD) research news to families and researchers around the world. From major clinical trial updates to scientific breakthroughs, we’ve been at the forefront, translating complex science into clear, understandable insights. But as HD research advances, so must we. Today, we’re launching our Spring Giving Campaign: "Hope in Full Bloom"—an opportunity for our community to come together and ensure that HDBuzz remains the trusted source for HD research news.

A Year of Growth and Impact

It’s been one year since HDBuzz transitioned to new leadership. In that time, we have:

* Doubled article output on critical HD research developments. If you feel like you’ve been seeing more HDBuzz content than ever before, you’re not imagining it! As the pace of HD research has accelerated, we’ve matched it. In past years, HDBuzz was churning out about 2 articles a month, and recently we’ve settled in to publishing about an article a week. But we’re not stopping there! HD researchers keep cranking out amazing science and clinical trial updates are on a roll, so we’re ramping up too! You may have noticed our Monday/Thursday publication schedule in March, which will be our new norm, so prep your inbox for HDBuzz emails twice a week moving forward.

* Expanded our team by bringing in new writers from top research institutions. As our output has grown, so has our writing team, offering readers perspectives from fresh voices within the HD research community. And we’re excited to expand this initiative too! We’re bringing back the HDBuzz Prize for Young Science Writers this summer, so stay tuned.

* Launched new social media channels (Instagram, Bluesky) to reach more people in new ways. The way people read and receive information is evolving, so we’re meeting people where they are—on new social media platforms. This will help us bring you our same great content in a fresh way while reaching a broader audience across multiple platforms to keep the global community up to date on HD research breakthroughs.

* Strengthened our funding model, raising over $23,000 through direct reader donations since October. Thank you! We’re still working toward financial independence and sustainability, but because of loyal readers and supporters, we’re headed there.


Every article, every update, every social media post represents our commitment to free, accurate, and accessible HD research news—because knowledge is power.

Why This Matters Now

2025 is shaping up to be a pivotal year in HD research!

Major clinical trials are reaching key milestones. The second quarter of this year will bring long-awaited data from multiple trials testing potential disease-modifying treatments. This includes both uniQure and PTC Therapeutics, who are both expected to report critical results by the end of June. Families, advocates, and researchers around the world will be looking for trusted, clear, and independent analysis—the kind of reporting that HDBuzz specializes in.

New discoveries are transforming the field. Researchers are learning more than ever about HD biology, from biomarkers that could get us to preventative trials to the reason behind why HD symptoms takes so long to show up. HDBuzz ensures these breakthroughs are translated into plain language, making cutting-edge science accessible to everyone.

The global HD community depends on accurate, unbiased information. With so much at stake, it’s critical that families have a source they can trust—one that cuts through jargon and provides clear, relevant updates.

The Challenge: Keeping HDBuzz Strong

While our impact is growing, so are the challenges of sustaining independent science journalism.

HDBuzz is funded entirely by donations from HD community organizations and readers. Unlike traditional media, we don’t sell ads or operate behind a paywall. We believe research news should be free to everyone, no matter their location or financial situation.

Importantly, HDBuzz has never accepted funding or support from drug companies. We love drug companies—we’re hoping they help us cure HD! But taking money from any organization dedicated to a particular therapy could give the impression of bias in our reporting, which we diligently aim to avoid.

Our goal is to raise $30,000 by May 27 to keep HDBuzz thriving, expand our reporting, and prepare for the biggest HD research updates of the year.

Here’s how you can help:

* Make a donation today—whether it’s a one-time gift or a monthly contribution. Every dollar fuels HD research news that remains free for all.

* Share our campaign with your network—on social media, in HD support groups, or with friends and family.

* Stay engaged—follow our updates, comment on our posts, and help amplify trusted HD research news.

* Follow us on social media and spread the word—like, share, and invite your friends and family to follow us too. We're on Facebook, Instagram, LinkedIn, and Bluesky. Every new follower helps expand awareness and support for HD research.


What Your Support Makes Possible

* $10/month helps us translate articles into multiple languages, reaching HD families worldwide.

* $50 supports real-time reporting from major HD research conferences.

* $200 funds an in-depth, expert-written article breaking down the latest scientific data.


If just 5% of our readers gave $20/month, we would be independently sustainable by the end of the year.

Join Us—Hope in Full Bloom for HD Research

Now is the time. With major trial results on the horizon, groundbreaking research happening now, and a growing community that relies on our reporting, we need your support more than ever.

Every donation, every share, and every engagement strengthens our ability to deliver trusted, timely, and independent HD research news.

Donate today to nurture knowledge, grow hope, and advance understanding of HD research.

Thank you for letting us be a part of your journey.

March 2025 was packed with groundbreaking discoveries in Huntington’s disease (HD) research, and we’re here to bring you the biggest highlights! From the cutting-edge CRISPR delivery system RIDE, which could rewrite the playbook on gene editing, to major advances in drug development, biomarker breakthroughs, and fresh insights into HD biology, this month was a whirlwind of progress. Scientists are pushing the boundaries of what’s possible, inching closer to real solutions for HD families. March not only brought us scientific progress, but also community events, like HDYO’s International Young Adult Congress and Factor-H’s Gratitude Day, that raise awareness about HD, provide a platform of support, and bring the community together. If you missed any of the exciting updates, don’t worry—we’ve got you covered. Dive into our March recap and catch up on all the HD news you need to know!

CHDI

Every year CHDI hosts one of the largest HD research conferences. The 2025 HD Therapeutics Conference showcased significant advancements in research and potential therapies for HD. Over three days, more than 400 scientific experts from around the world convened to discuss clinical trial updates, genetic modifiers, and innovative technologies.

Day 1: Progress in Clinical Trials

The conference began with updates on therapeutic trials targeting huntingtin (HTT), the protein responsible for HD. Researchers shared data on ongoing efforts to reduce HTT levels safely, including small molecule drugs and gene therapies. Discussions also focused on refining clinical trial design, selecting meaningful biomarkers, and ensuring that future trials are better equipped to detect therapeutic benefits.

Day 2: Genetic Modifiers and Disease Progression

The second day explored genetic factors that influence when HD symptoms begin and how the disease progresses. Scientists presented findings from large-scale genetic studies identifying key modifiers that may delay disease onset, offering promising new therapeutic targets. Advances in understanding DNA repair pathways and their role in HD progression were also highlighted, providing new directions for potential treatments.

Day 3: New Technologies and Future Directions

The final day spotlighted cutting-edge technologies that could revolutionize HD research and treatment. Talks covered advances in gene editing, innovative drug delivery methods, and AI-driven approaches to analyzing HD progression. Researchers emphasized the importance of collaboration and continued innovation to translate these discoveries into real-world therapies.

The conference reinforced the growing momentum in HD research, with scientists and industry partners working together to turn breakthroughs into meaningful treatments for families affected by HD.

Wake up call: Sleep is impacted before Huntington’s disease symptoms appear

A recent study, highlighted during Sleep Awareness Week, reveals that sleep disturbances can occur in individuals carrying the HD gene up to 15 years before the onset of other symptoms. Researchers observed that those less than 15 years from predicted symptom onset experienced fragmented sleep and increased nighttime wakefulness, while those more than 15 years away may not show significant sleep issues. These findings suggest that early sleep disruptions may contribute to thinking and mood impairments associated with HD, highlighting the potential of sleep-focused interventions to improve quality of life and possibly slow disease progression.

Listening for Whispers: How a Tiny Protein Could Transform HD Research

A recent 14-year longitudinal study has demonstrated that neurofilament light (NfL), a protein released by damaged brain cells, can serve as an early indicator of HD progression. Elevated NfL levels were detected in individuals carrying the HD gene many years before the onset of symptoms, correlating with disease advancement. This finding suggests that monitoring NfL through simple blood tests could revolutionize HD research by predicting symptom onset, enhancing clinical trial design, and enabling earlier therapeutic interventions to potentially slow or halt disease progression.

The Huntington’s Disease Youth Organization’s World Congress: Supporting Young People Affected by HD

The Huntington’s Disease Youth Organization (HDYO) supports, educates, and empowers young people affected by HD. HDYO’s recent biennial International Young Adult Congress provided a unique space for connection and learning. At the 2025 Congress in Prague, attendees engaged in workshops, heard from leading researchers, and shared personal experiences, fostering a strong sense of community.

HDBuzz gave two presentations, firstly setting the stage with HD research terminology 101, then diving into an overview of important research going on at the benchside and in the clinic. Key themes in other talks included mental health, genetic testing decisions, and navigating family dynamics, with experts offering guidance tailored to young people facing HD. The event underscored the importance of youth involvement in research, advocacy, and peer support, reinforcing HDYO’s mission to empower the next generation of HD families.

Gratitude Day

Factor-H is a nonprofit dedicated to supporting HD families in Latin America, where extreme poverty and lack of resources make the disease even more devastating. This month, HDBuzz caught up with Factor-H founder, Dr. Ignacio Muñoz-Sanjuán, in an interview that details the unique problems that people with HD there face, and how they help and support these vulnerable families.

The organization provides humanitarian aid, medical care, housing improvements, education, caregiver training, and legal advocacy to some of the most vulnerable HD communities in Venezuela, Peru, and Colombia. These same communities played a crucial role in the discovery of the HD gene in 1993, yet many still lack basic necessities. Factor-H also works to combat stigma, educate the public, and advocate for sustainable, long-term support.

A key initiative is Gratitude Day, held this year on March 23rd, which honors HD families’ contributions to research while raising awareness of their ongoing struggles. The 2025 event included a live stream from Venezuela, candlelight vigils, and medical outreach, reinforcing the importance of global solidarity. Factor-H’s mission highlights the need for both immediate relief and systemic change, ensuring that hope and humanity—not just hardship—define the future for HD families.

Molecular Surgeons for Huntington’s Disease Catch a RIDE with CRISPR Advancements

A groundbreaking gene-editing technology called RIDE (Ribonucleoprotein Delivery) is showing promise for treating HD. RIDE uses CRISPR, a powerful tool to edit DNA, and delivers it precisely to targeted cells in the brain, overcoming key challenges like off-target effects and immune responses. Early tests in mice and monkeys have demonstrated that RIDE can effectively reduce harmful HTT protein levels in the brain, leading to improved behavior and motor function. This system offers lasting effects from a single injection and is designed to minimize the risks of unintended genetic changes. While still in early stages, RIDE's targeted approach brings new hope for HD treatments, with the potential for broader applications in genetic diseases.

March was an exciting month for HD research, filled with groundbreaking discoveries, inspiring community events, and promising advancements toward better treatments. We hope you enjoyed this recap and feel as energized as we do about the increased pace of progress being made. Science is moving fast, and every step forward brings us closer to real treatments for HD families. Stay tuned to HDBuzz for the latest updates in HD research, and as always, thank you for being part of this journey with us!

Imagine a tiny, microscopic surgeon moving through the body, making precise genetic repairs exactly where they’re needed. That’s the vision behind a groundbreaking new gene-editing delivery system called RIDE—Ribonucleoprotein Delivery—recently featured in Nature Nanotechnology. This system offers a novel way to deliver CRISPR, a powerful gene-editing tool, to specific cells in the body. Researchers have tested RIDE in mice and monkeys, with promising results for diseases like Huntington’s disease (HD). Let’s dive into how this innovative system could shape the future of HD treatments.

CRISPR Challenges

CRISPR has revolutionized the way scientists approach genetic diseases, acting as a molecular “find and replace” tool for DNA. For HD, where an expanded CAG repeat in the huntingtin (HTT) gene leads to breakdown of brain cells, CRISPR could potentially correct or silence the faulty gene. However, several key challenges have stood in the way:

* Off-Target Effects – CRISPR must be highly precise to avoid accidental edits in unintended parts of the genetic code.

* Immune System Response – The body may recognize the CRISPR components as foreign invaders and attack them.

* Targeted Delivery – The therapy must reach the right cells in the brain, specifically nerve cells in the very center of the brain that’s most affected in HD, without affecting other tissues.


RIDE aims to overcome these hurdles by packaging CRISPR into engineered particles that have been given brain-specific navigation systems that lets them go to specific cell types.

How RIDE Works

RIDE delivers CRISPR in the form of ribonucleoproteins , which are pre-formed complexes of the editing enzyme and guide RNA—think of this like the genetic navigation system that directs RIDE exactly where to go. The complex is packaged inside a virus-like particle, which acts as a protective car. These particles can be engineered with molecular tags, similar to putting a precise street address in your car’s navigation system, so that they target specific cells, ensuring precise delivery.

The key innovation of RIDE lies in its ability to be customized for different cell types. By modifying the outer shell of these nanoparticles with specific molecular tags, scientists can direct them toward desired cells, ensuring that the CRISPR machinery reaches the correct targets. This is a big leap forward for CRISPR-based gene editing.

To visualize the process, imagine a package being delivered. Current CRISPR approaches can make deliveries to the right neighborhood, but RIDE is a door-to-door service that drops the package at the exact address. RIDE’s customization allows the CRISPR machinery to be sent exactly where it’s needed, reducing off-target effects and improving efficiency.

Testing RIDE for HD

Researchers tested RIDE in mice that model HD, focusing on neurons in the striatum—the central brain region most affected by HD. The goal was to silence or edit the mutated HTT gene that causes HD to slow or stop disease progression.

The results were striking: treated mice showed a reduction in HTT protein levels and improved behavior compared to untreated HD mice, such as their movement control on obstacle courses. Importantly, the editing efficiency was much higher than what has been achieved with other delivery methods that don’t have a cellular navigation system, and there were fewer signs of unwanted genetic changes or immune responses.

Another promising aspect of RIDE is its potential to provide long-lasting effects. All the results in these mice were achieved with just a single injection of RIDE; they didn't need to continuously administer the treatment. They monitored the mice for over 110 days (which is quite a long time in a mouse's lifespan!), and the improvements persisted.

An important safety component of the RIDE system is that the gene-editing tools are delivered as an assembled complex rather than as genetic material. This means that it’s only around for a short time, long enough to do its job, before being quickly degraded by the cell’s natural processes—another big leap forward for this CRISPR-based gene editing approach. This reduces the risk of persistent off-target effects that could arise from prolonged CRISPR activity.

Beyond Mice

To really assess the potential of RIDE for treating HD in humans, the researchers needed to move beyond mice. That's where the monkey studies come in. These studies help get a better understanding of how RIDE might behave in a system that's more closely related to humans.

Safety was the top priority considering this treatment involves injecting something directly into the brain. They used MRI scans to look for any signs of brain damage after a RIDE injection, and they didn't find any. They analyzed brain tissue samples and found that RIDE was in fact able to reduce levels of the non-expanded HD protein in the targeted areas. So they're seeing consistent results in both mice and monkeys, which is a good sign.

The researchers also went a step further to confirm that RIDE could work in human cells. To do this, they used stem cells that they turned into neurons. These studies checked many of the boxes that suggested this technology is working: they were able to target and edit the HD gene and there were surprisingly minimal off-target effects. So they got the green light for RIDE in a human-based model as well.

What This Means for HD Families

While these results are promising, RIDE is still in early stages of development. Further studies in larger animals, and eventually human trials, will be necessary before this approach can be considered for clinical use.

However, this study is a major step forward in the field of gene therapy. It demonstrates that RIDE has the potential to be a safe, effective, and highly targeted method for delivering CRISPR-based drugs. And the researchers are already exploring ways to expand RIDE's capabilities, including the potential to use systemic delivery.

Systemic delivery could mean scientists would be able to inject RIDE into the bloodstream and have it reach cells within the brain. This would be incredible, and a game-changer for HD and many other genetic diseases. Research breakthroughs around CRISPR are moving quickly—this technology has the potential to change the landscape of medicine as we know it.

Looking Ahead

RIDE represents an exciting step forward in the search for effective HD treatments. By combining the precision of CRISPR with an advanced delivery system that targets specific cell types and shuts off after editing, scientists are getting closer to making genetic therapies for HD a reality. And this team isn’t the only one working on this type of approach.

While we’re still in the early stages of this research, this breakthrough brings new hope to the HD community. This study offers a glimpse into a future where we might be able to treat genetic diseases, like HD, with unprecedented precision and effectiveness.
Stay tuned for more updates on how gene-editing technologies like RIDE continue to evolve and push the boundaries of what’s possible in HD research.