A Treatment for Huntington’s Disease

Last week the BBC reported on a successful clinical trial for a gene therapy to treat the progression of Huntington’s Disease. 

The paper published in November 2024 (restricted access) describes the methods of delivering a micro-RNA that targets the HTT gene via a viral vector. This gene therapy is called AMT-130. This paper described promising interim results of a high dose of AMT-130 resulting in an 18% reduction of disease progression when compared to a historical control group.

Huntington’s Disease Clinical Trials Update: September 2024

In this edition of the Huntington’s Disease Clinical Trials Update, we expand on the ongoing extension study of PTC518 from PTC Therapeutics, including 12-month interim results from the parent stu…

PaperstarsMena Farag, Sarah J Tabrizi, Edward J Wild

In the follow-up paper published in May 2025 (open access), further details of the treatment were given and discussions on accelerated approval with the FDA and other organisations. 

Huntington’s disease clinical trials update: March 2025

In this edition of the Huntington’s Disease Clinical Trials Update, we expand on the ongoing phase I clinical trial of ALN-HTT02 from Alynlam Pharmaceuticals. We also report on the SAGE-718 (also k…

PaperstarsMena Farag, Sarah J Tabrizi, Edward J Wild

The timelines of these papers and the ones preceding them from this group, dating back to 2012, illustrate process of discovery through to clinical trials, and in this case with incredibly promising results!