Advancing Huntington’s Disease Research: Harness at the 21st Annual CHDI Foundation Therapeutics Conference

Harness’s Jan Thirkettle, Madeleine Wakeling, and Andy Billinton have just returned from an invaluable week in Palm Springs, California, where they attended the 21st Annual CHDI Huntington’s Disease Therapeutics Conference.

This conference brought together leading researchers, clinicians, drug developers, advocates and families, with a strong emphasis on collaboration to address CHDI’s mission: accelerating the development of disease-modifying therapeutics for Huntington’s disease (HD). The incredible pace and breadth of progress across a wide range of topics was evident. For our team, just a few of the stand-out themes were:

· The continued progress on somatic expansion biology Steve McCarroll shared his team’s latest work on disease pathology at a cellular level; and Jang-Ho Cha shared Latus’ work on disease modelling which predicts that slowing somatic expansion could delay disease onset by decades.
· Meetings with patient advocacy organisations such as Huntington's Disease Youth Organization (HDYO) and Huntington's Disease Society of America (HDSA), which provided further insights into community interactions and barriers to participation in clinical research.
· The latest GWAS analysis presented by Jim Gusella, Sahar Gelfman and Qingqin Li which provided further insight into the importance of FAN1 as a modifier of somatic expansion across a range of symptom measures and disease stages.

This year we had the privilege to be able to share our progress on our FAN1 programme as part of a session on somatic expansion and the therapeutic potential of DNA-repair modifiers in HD. Andy presented our work over the past 3 years; from target validation to development of our MISBA® platform for controlled upregulation and most recently, the selection of HRN001, our drug candidate for HD. We were thrilled by the engagement and left with many more friends and potential collaborators to follow up with.

Harness remains committed to working with the scientific and patient communities to accelerate progress toward meaningful therapies for HD.

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