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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

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

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

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

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

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

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

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

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

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

Next is Dr. Christopher Mezias from the Critical Path Institute, who will discuss frameworks for regulatory science and biomarker validation. Standardizing biomarker assays and benchmarks is key to accelerating approval of HD therapies. #HDCongress2025

He also highlights that they’re moving forward with development plans and hope to start a clinical trial in 2027. #HDCongress2025

Blair touches on some of the elements of research that are more often presented to investors - considerations around intellectual property and plans for manufacturing. These factors are important as young companies seek investments in early stage clinical studies! #HDCongress2025

IG-HD01 is an allele-selective gene editor, meaning that in each cell, it chops out a portion of the copy of huntingtin containing the CAG repeat expansion, while leaving the healthy copy intact. Of note, this means that Incisive’s technology targets DNA, not the mRNA copy message. #HDCongress2025

He believes that targeting the DNA, the source of the expanded HD-causing protein, should be the most efficient way to intervene in the toxic pathways leading to HD symptoms. #HDCongress2025

Blair is introducing Incisive’s therapeutic “pipeline” laying out methods, biological targets, and plans for trying to move their drugs into the clinic. He is focusing today on IG-HD01, their “lead candidate” (furthest developed drug) for HD. #HDCongress2025

Blair is showing this with beautiful fluorescent images. They have also examined different aspects of safety and toxicity. #HDCongress2025

Incisive has done a variety of experiments to show that their methods lead to efficient delivery of gene editing technology, in cells as well as in different tissues in animal models. #HDCongress2025

You can think of LNPs like micro Trojan horses - they contain the therapeutic drugs against HD and the LNP gets them to where we want them to be. #HDCongress2025