There are few neurodegenerative diseases as devastating as Huntington’s. It’s sometimes likened to having Parkinson’s, ALS and Alzheimer’s all at the same time, with symptoms that include progressive motor dysfunction, cognitive decline and behavioural change. It’s also hereditary — if a person has the faulty gene that causes the disease, there’s a 50 percent chance their children will have it, too. In the fall of 2025, however, scientists announced that, for the first time, they could reduce the progression of Huntington symptoms using a new gene therapy. While that clinical breakthrough came with several caveats, it also heralded a possible new paradigm for drug discovery. In this episode, we explore how this innovative therapy works and what it could mean for the treatment of other rare diseases. 

Featured in this episode:

Rachel Harding is an assistant professor in the department of pharmacology and toxicology at the University of Toronto and a principal investigator at the Structural Genomics Consortium. Her work on Huntington’s disease has been recognized with major early-career awards, highlighting both scientific excellence and the potential patient impact of her research program.

Further reading:

Research is unravelling the mystery of what causes Huntington’s disease, a devastating brain disease

In a first, a gene therapy seems to slow Huntington disease

“Best news” for Huntington’s disease community comes with unanswered questions

The Huntington’s disease research pipeline

World’s first patient treated with personalized CRISPR gene editing therapy at Children’s Hospital of Philadelphia

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