Up next is HDBuzz’s own Rachel Harding! When she’s not Buzzing away, Rachel studies how the HD-related expansion of the huntingtin protein affects its structure and its function. #HDTC2026
February 24, 2026 at 5:43 PM
Up next is HDBuzz’s own Rachel Harding! When she’s not Buzzing away, Rachel studies how the HD-related expansion of the huntingtin protein affects its structure and its function. #HDTC2026
Good morning! HDBuzz is at CHDI's Huntington's Disease Therapeutics Conference, ready to bring you live updates from the most recent research in the field. Get ready for some exciting science! #HDTC2026
February 24, 2026 at 4:38 PM
Good morning! HDBuzz is at CHDI's Huntington's Disease Therapeutics Conference, ready to bring you live updates from the most recent research in the field. Get ready for some exciting science! #HDTC2026
We are back for the third and final day of #HDTC2026! This morning’s session focuses on new and breakthrough science both in and out of the HD field that is driving therapeutic development.
February 26, 2026 at 5:08 PM
We are back for the third and final day of #HDTC2026! This morning’s session focuses on new and breakthrough science both in and out of the HD field that is driving therapeutic development.
First up is Taekjip Ha from Boston Children’s Hospital and Harvard Medical School. He zooms waaaaay inside cells to look at single molecules so that he can track what’s going on in somatic expansion in HD. #HDTC2026
February 26, 2026 at 5:08 PM
First up is Taekjip Ha from Boston Children’s Hospital and Harvard Medical School. He zooms waaaaay inside cells to look at single molecules so that he can track what’s going on in somatic expansion in HD. #HDTC2026
To answer this question, Gregory looked at what’s done in nature. Inside the cell, there are proteins that span the cell’s membrane. Interestingly they all have the same shape - a spiral looking thing called an alpha helix. #HDTC2026
February 26, 2026 at 6:59 PM
To answer this question, Gregory looked at what’s done in nature. Inside the cell, there are proteins that span the cell’s membrane. Interestingly they all have the same shape - a spiral looking thing called an alpha helix. #HDTC2026
Now TJ is showing us some cool movies of the bending DNA “dancing” around. Although we often think of these molecules as static, they are actually in constant motion, and the movement of DNA can impact how it is repaired. #HDTC2026
February 26, 2026 at 5:21 PM
Now TJ is showing us some cool movies of the bending DNA “dancing” around. Although we often think of these molecules as static, they are actually in constant motion, and the movement of DNA can impact how it is repaired. #HDTC2026
Taekjip’s team also looks at how these machines might bend or contort the DNA. They ask how the bending might be altered with CAG DNA bubbles of different shapes and sizes, and when different repair machines work to fix them. #HDTC2026
February 26, 2026 at 5:16 PM
Taekjip’s team also looks at how these machines might bend or contort the DNA. They ask how the bending might be altered with CAG DNA bubbles of different shapes and sizes, and when different repair machines work to fix them. #HDTC2026
To overcome this potential safety concern, they will test a 2-virus approach in animal models. The room is buzzing about this promising therapeutic avenue, and you can be sure that a roomful of 450 HD researchers will take on the challenge! #HDTC2026
February 26, 2026 at 6:35 PM
To overcome this potential safety concern, they will test a 2-virus approach in animal models. The room is buzzing about this promising therapeutic avenue, and you can be sure that a roomful of 450 HD researchers will take on the challenge! #HDTC2026
Thus far, a lead candidate discovered in this way is having "extraordinary clinical responses.” It just received fast track approval for a wide array of cancers driven by a particular gene. This could be available by the FDA as early as 2027 - encouraging! #HDTC2026
February 26, 2026 at 7:05 PM
Thus far, a lead candidate discovered in this way is having "extraordinary clinical responses.” It just received fast track approval for a wide array of cancers driven by a particular gene. This could be available by the FDA as early as 2027 - encouraging! #HDTC2026
This will hopefully further strengthen uniQUre’s case with the FDA (en.hdbuzz.net/uniqure-secu...) to move towards accelerated approval. #HDTC2026
https://en.hdbuzz.net/uniqure-secures-type-a-meeting-with-fda-what-this-means-for-amt-130/]
February 24, 2026 at 5:11 PM
This will hopefully further strengthen uniQUre’s case with the FDA (en.hdbuzz.net/uniqure-secu...) to move towards accelerated approval. #HDTC2026
They have tested their drug in different types of cells in a dish, including cells from people with Friedreich's Ataxia. The drug seems to be working as expected and changing gene expression. Now they are interested in applying this technology and approach to target other repeat diseases. #HDTC2026
February 26, 2026 at 7:45 PM
They have tested their drug in different types of cells in a dish, including cells from people with Friedreich's Ataxia. The drug seems to be working as expected and changing gene expression. Now they are interested in applying this technology and approach to target other repeat diseases. #HDTC2026
And we are back for Day 2 of #HDTC2026 ! This morning, we are focussed on research seeking to understand what is really driving Huntington’s disease (HD) using new genetic tools and technologies that can look at the detailed molecular changes that happen through HD. Grab your coffee and settle in!
February 25, 2026 at 5:05 PM
And we are back for Day 2 of #HDTC2026 ! This morning, we are focussed on research seeking to understand what is really driving Huntington’s disease (HD) using new genetic tools and technologies that can look at the detailed molecular changes that happen through HD. Grab your coffee and settle in!
He is sharing several vignettes from his lab - experimental stories that he hopes will lead to answers around questions in the HD field. #HDTC2026
February 26, 2026 at 10:19 PM
He is sharing several vignettes from his lab - experimental stories that he hopes will lead to answers around questions in the HD field. #HDTC2026
Gregory is interested in the multiple interactions of the ASO package and how it’s bound at the molecular level in intricate detail, to improve drug design. #HDTC2026
February 26, 2026 at 7:19 PM
Gregory is interested in the multiple interactions of the ASO package and how it’s bound at the molecular level in intricate detail, to improve drug design. #HDTC2026
Gregory is even doing cross-species experiments in his role at the company LifeMine, where they’re de-coding the genetics of fungi to find advancements for “undruggable” targets. #HDTC2026
February 26, 2026 at 7:14 PM
Gregory is even doing cross-species experiments in his role at the company LifeMine, where they’re de-coding the genetics of fungi to find advancements for “undruggable” targets. #HDTC2026
They can test how well connected the neurons are by adding different signalling molecules and measuring how efficiently electrical and chemical signals are transmitted through the mini brains. #HDTC2026
February 26, 2026 at 5:46 PM
They can test how well connected the neurons are by adding different signalling molecules and measuring how efficiently electrical and chemical signals are transmitted through the mini brains. #HDTC2026
Most current disease treatments are designed to target protein molecules and change how they work. Aseem is interested in targeting drugs to genetic material (DNA and RNA) instead. #HDTC2026
February 26, 2026 at 7:29 PM
Most current disease treatments are designed to target protein molecules and change how they work. Aseem is interested in targeting drugs to genetic material (DNA and RNA) instead. #HDTC2026
Biological molecules can be “undruggable” for different reasons, like “flatness,” difficulty of two molecules bonding, or energy. Gregory’s group has overcome many of these challenges by turning to nature. #HDTC2026
February 26, 2026 at 7:13 PM
Biological molecules can be “undruggable” for different reasons, like “flatness,” difficulty of two molecules bonding, or energy. Gregory’s group has overcome many of these challenges by turning to nature. #HDTC2026
After a quick coffee break, our next speaker is Gregory Verdine from Harvard. He’s hoping to open our eyes to a new avenue of drug discovery. A lofty goal! #HDTC2026
February 26, 2026 at 6:52 PM
After a quick coffee break, our next speaker is Gregory Verdine from Harvard. He’s hoping to open our eyes to a new avenue of drug discovery. A lofty goal! #HDTC2026
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!
February 27, 2026 at 1:08 AM
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!
That’s all for this morning. We are going to lunch and to cool off before we get back with updates on the final session of talks this afternoon. Stay tuned. #HDTC2026
February 26, 2026 at 8:19 PM
That’s all for this morning. We are going to lunch and to cool off before we get back with updates on the final session of talks this afternoon. Stay tuned. #HDTC2026
He’s bravely sharing what scientists call “negative data” - meaning that some of the experiments they designed didn’t work, but they informed future ones so they still provide very valuable information. #HDTC2026
February 24, 2026 at 6:23 PM
He’s bravely sharing what scientists call “negative data” - meaning that some of the experiments they designed didn’t work, but they informed future ones so they still provide very valuable information. #HDTC2026
TJ also describes a new technology that is a better way to read the DNA sequence around the CAG repeat in the huntingtin gene. Those CAG repeats can be pesky to measure! #HDTC2026
February 26, 2026 at 5:28 PM
TJ also describes a new technology that is a better way to read the DNA sequence around the CAG repeat in the huntingtin gene. Those CAG repeats can be pesky to measure! #HDTC2026
Some of the data from people who received AMT-130 wasn’t compared to a placebo group, but rather participants in ENROLL-HD, an observational study that follows people with HD over time. #HDTC2026
February 24, 2026 at 4:46 PM
Some of the data from people who received AMT-130 wasn’t compared to a placebo group, but rather participants in ENROLL-HD, an observational study that follows people with HD over time. #HDTC2026