A new system has been developed that allows researchers to fine-tune gene expression with oral drugs, work that provides a powerful tool for gene editing.
Voyager Therapeutics is shifting towards a new technology to deliver gene therapy and away from a planned HD clinical trial. But this could lead to less invasive drugs in the long run, and many other companies are working on HD gene therapies.
A new study by researchers at Johns Hopkins describes a non-invasive way to track progression of Huntington’s disease. This could be used before patients even start showing symptoms to help test treatments in early stages of disease.
A successful early trial of a drug for Familial Transthyretic Amyloidosis showed that CRISPR gene editing could be safely used in the human body. What does this mean for gene editing in HD?
New studies pinpoint precisely how the drug pridopidine works in models of Huntington’s disease
Catch up on all of the latest updates from day 3 of the 2021 CHDI Huntington’s disease therapeutics conference #HDTC2021
Catch up on all of the latest updates from day 1 of the 2021 CHDI Huntington’s disease therapeutics conference #HDTC2021 including presentations from Wave, Roche and Triplet.
Catch up on all of the latest updates from day 2 of the 2021 CHDI Huntington’s disease therapeutics conference #HDTC2021
Catch up on all the latest Huntington's disease clinical trial news in this one stop shop article covering all of the recent developments in making medicines for Huntington's disease
The first group of 10 participants have been dosed in uniQure’s clinical trial of an HD gene therapy, and three new manuscripts describe safe, widespread huntingtin lowering in animals.
Wave Life Sciences shared the disappointing news that their two ASOs in Phase 1/2 trials in HD patients did not successfully lower mutant huntingtin.