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Huntington’s disease research news. In plain language. Written by scientists. hdbuzz.net
Editors - @sjh508 @rjharding
Editors - @sjh508 @rjharding
That’s all for us from the #HDCongress2025! We hope you enjoyed the coverage and we’ll see you next year!
October 13, 2025 at 9:36 PM
That’s all for us from the #HDCongress2025! We hope you enjoyed the coverage and we’ll see you next year!
Next steps for their company involve preparing to submit an IND (investigational new drug) application with the FDA, the first step that tells regulators about plans to move towards clinical trials in humans. #HDCongress2025
October 13, 2025 at 9:34 PM
Next steps for their company involve preparing to submit an IND (investigational new drug) application with the FDA, the first step that tells regulators about plans to move towards clinical trials in humans. #HDCongress2025
Latus has data to show that their MSH3-targeting virus can reduce the expansion of CAG repeats in HD mouse models - the higher the dose, the more it reduces somatic instability. #HDCongress2025
October 13, 2025 at 9:33 PM
Latus has data to show that their MSH3-targeting virus can reduce the expansion of CAG repeats in HD mouse models - the higher the dose, the more it reduces somatic instability. #HDCongress2025
In people, tiny changes in MSH3 can dictate how early or late HD symptoms appear. In different models, knocking out MSH3 has slowed the expansion of CAG repeats and led to improvements in cell health and behavior. #HDCongress2025
October 13, 2025 at 9:33 PM
In people, tiny changes in MSH3 can dictate how early or late HD symptoms appear. In different models, knocking out MSH3 has slowed the expansion of CAG repeats and led to improvements in cell health and behavior. #HDCongress2025
So they’ve got this very effective “envelope” that can be delivered to the right place, and inside it they put a piece of man-made genetic code that can target a DNA repair gene called MSH3. #HDCongress2025
October 13, 2025 at 9:29 PM
So they’ve got this very effective “envelope” that can be delivered to the right place, and inside it they put a piece of man-made genetic code that can target a DNA repair gene called MSH3. #HDCongress2025
Jang-Ho is showing fluorescent images demonstrating that their virus can enter and spread from the deep brain areas that drive changes in movement and motivation in HD, and outward to the areas involved in cognition and executive function. #HDCongress2025
October 13, 2025 at 9:28 PM
Jang-Ho is showing fluorescent images demonstrating that their virus can enter and spread from the deep brain areas that drive changes in movement and motivation in HD, and outward to the areas involved in cognition and executive function. #HDCongress2025
Latus has engineered specialized, harmless viruses (AAVs) to deliver genetic drugs to brain cells - they are specifically focused on ways to do this in the right location and at low doses. Historically it has been very difficult to get these viruses to areas deep inside the brain. #HDCongress2025
October 13, 2025 at 9:27 PM
Latus has engineered specialized, harmless viruses (AAVs) to deliver genetic drugs to brain cells - they are specifically focused on ways to do this in the right location and at low doses. Historically it has been very difficult to get these viruses to areas deep inside the brain. #HDCongress2025
Latus targets the brain areas most affected by the diseases they study, which for HD is the deep brain structures known as the striatum. #HDCongress2025
October 13, 2025 at 9:26 PM
Latus targets the brain areas most affected by the diseases they study, which for HD is the deep brain structures known as the striatum. #HDCongress2025
As a neurologist, Jang-Ho reminds the crowd that medicine in neurology is driven by 3 rules - “location, location, location”. In other words, for a gene therapy to work, it has to hit the right part of the brain and it has to be distributed in an efficient way. #HDCongress2025
October 13, 2025 at 9:26 PM
As a neurologist, Jang-Ho reminds the crowd that medicine in neurology is driven by 3 rules - “location, location, location”. In other words, for a gene therapy to work, it has to hit the right part of the brain and it has to be distributed in an efficient way. #HDCongress2025
Latus was founded by Dr. Bev Davidson, a world leader in gene therapy and HD research. They work on one-and-done gene therapy treatments for serious brain diseases, and lots of their leadership have a background in the HD field. #HDCongress2025
October 13, 2025 at 9:24 PM
Latus was founded by Dr. Bev Davidson, a world leader in gene therapy and HD research. They work on one-and-done gene therapy treatments for serious brain diseases, and lots of their leadership have a background in the HD field. #HDCongress2025
The final talk of the conference is from Dr. Jang-Ho Cha of Latus Bio, who will present data on targeting MSH3 to prevent CAG repeat expansion, thought to be one of HD’s root causes. #HDCongress2025
October 13, 2025 at 9:21 PM
The final talk of the conference is from Dr. Jang-Ho Cha of Latus Bio, who will present data on targeting MSH3 to prevent CAG repeat expansion, thought to be one of HD’s root causes. #HDCongress2025
One of C-Path’s goals is to make sure that measurements made across many locations using diverse technology (like different MRI machines) will be consistent enough across the board to use in a clinical trial. When there’s a lot of variation, that requires a closer look. #HDCongress2025
October 13, 2025 at 9:18 PM
One of C-Path’s goals is to make sure that measurements made across many locations using diverse technology (like different MRI machines) will be consistent enough across the board to use in a clinical trial. When there’s a lot of variation, that requires a closer look. #HDCongress2025
Chris highlights the complexity of approaching regulators like the FDA with a new biomarker to use in clinical trials, which requires providing evidence on its usefulness, in what context it will be used, and what it adds to the field. #HDCongress2025
October 13, 2025 at 9:18 PM
Chris highlights the complexity of approaching regulators like the FDA with a new biomarker to use in clinical trials, which requires providing evidence on its usefulness, in what context it will be used, and what it adds to the field. #HDCongress2025
C-Path uses a framework to make decisions about what aspects of collaboration, data, and drug development to prioritize. It incorporates perspectives from many “stakeholders” including family members, patient facing orgs, regulators, scientists, clinicians, and companies. #HDCongress2025
October 13, 2025 at 9:15 PM
C-Path uses a framework to make decisions about what aspects of collaboration, data, and drug development to prioritize. It incorporates perspectives from many “stakeholders” including family members, patient facing orgs, regulators, scientists, clinicians, and companies. #HDCongress2025
Chris shared this slide, summarizing the complexities of biomarkers. #HDCongress2025
October 13, 2025 at 9:14 PM
Chris shared this slide, summarizing the complexities of biomarkers. #HDCongress2025
Chris touches on the many categories of biomarkers and the complexity of how they are used to focus on disease progression, treatments, and response. CHDI and C-Path recently held a workshop to discuss how best to use imaging as a biomarker for HD progression. #HDCongress2025
October 13, 2025 at 9:12 PM
Chris touches on the many categories of biomarkers and the complexity of how they are used to focus on disease progression, treatments, and response. CHDI and C-Path recently held a workshop to discuss how best to use imaging as a biomarker for HD progression. #HDCongress2025
There are different ways to get a new biomarker to be accepted by a regulatory agency like the FDA as an endpoint in a clinical trial. These are formal processes that have to be approached in collaboration with researchers, companies, and affected communities. #HDCongress2025
October 13, 2025 at 9:10 PM
There are different ways to get a new biomarker to be accepted by a regulatory agency like the FDA as an endpoint in a clinical trial. These are formal processes that have to be approached in collaboration with researchers, companies, and affected communities. #HDCongress2025
Chris redefines a biomarker, something we can measure to track disease and determine how treatments are working, and reminds us that many approaches to tracking HD are necessary at different stages of the disease. #HDCongress2025
October 13, 2025 at 9:08 PM
Chris redefines a biomarker, something we can measure to track disease and determine how treatments are working, and reminds us that many approaches to tracking HD are necessary at different stages of the disease. #HDCongress2025
The HD-RSC (Regulatory Science Consortium) is a partnership between the Critical Path Institute, an organization that brings people together in various disease spaces, and other organizations, like HD nonprofits, companies, and the FDA. #HDCongress2025
October 13, 2025 at 9:06 PM
The HD-RSC (Regulatory Science Consortium) is a partnership between the Critical Path Institute, an organization that brings people together in various disease spaces, and other organizations, like HD nonprofits, companies, and the FDA. #HDCongress2025