Charles Sabine talks us through the highlights from the CHDI conference that took place between 26 - 29 February 2024.


This annual CHDI conference is a special event for the Huntington's community that brings researchers together for three days of innovation, inspiration and hope. 

This year, 55 pharmaceutical and biotech companies attended the conference, many of which are close to launching clinical trial data.

PTC Therapeutics discussed their splicing study which aims to decrease the amount of Huntingtin protein that is made. It does this to both the normal gene and the mutated gene and would be taken in pill form. They are expecting to be sharing some more data from the study in the coming month.

Wave Life Sciences spoke to Charles about the single-dose data from their SELECT-HD study that could have a significant reduction of the Huntingtin mutation with results from the multi-dose study due later this year.

uniQure said:

It's really exciting being here at this meeting and to see the wealth of data and to get a better understanding of the underlying biology of the disease. So I would imagine there is potential opportunity for new therapies to merge and I think there will be an opportunity to combine treatment or to have people on multiple different therapies at the same time. 

Charles speaks to a new company, Atalanta Therapeutics who are looking at a new way to lower the Huntingtin gene. Their drug would be administered by injection and although yet to be tested on humans, they expect the drug to work and keep the protein lowered for at least six months.

He discusses the CAG repeat length with Vanessa Wheeler PHD from Massachusettes General Hospital and Harvard Medical School and her optimistic views on how we could slow the repeat expansion down.

We also learn about somatic instability and how when the CAG repeats continue to grow, repair pathways in the Huntingtin gene try to fix the problem but ultimately it makes things worse. Rgenta Therapeutics are looking at fixing the mismatch repair pathway to make it function how it's supposed to.

Rgenta Therapeutics said:

Our goal is to push this as rapidly as we can, and part of the reason that we're out here at CHDI's conference is to connect with the leaders across this spectrum, clinical, pre-clinical and biomarker, so that we can accelerate that even further.It is a phenomenal conference. I think the CHDI community is really something to be admired, from early researchers trying to identify what target or gene could we go after to help fix the disease, to clinicians who are treating patients, it's a full spectrum of amazing people.

Roche finally discuss what they have learnt from past trials and how they believe trials in the future could be conducted more quickly and with fewer participants. They also talk about working more closely with Enroll-HD which is the world's largest research recruitment platform that anyone from the Huntington's disease community can join and they currently have over 21,000 participants.

With pharmaceutical companies known within the community such as uniQure and Roche to new start-up companies looking into innovative ways of combatting Huntington's disease. Both of these rely on the participation of Huntington's disease patients and families across the globe. We want to say thank you to those who commit their time to helping us get one step closer to finding new treatments.

You can watch the full video below.


Now, we, Huntington's families including young people are the crucial element. The more we engage with this exciting research, the sooner we'll have new treatments.

- Charles Sabine