Latest news

Another tool in the box: Creation of a molecular “dimmer switch” advances gene editing

Another tool in the box: Creation of a molecular “dimmer switch” advances gene editing

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.

Dr Leora FoxAugust 30, 2021

Unpacking recent gene therapy press

Unpacking recent gene therapy press

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.

Dr Leora Fox and Dr Sarah HernandezAugust 16, 2021

Does blood hold the key to testing treatments earlier in HD patients?

Does blood hold the key to testing treatments earlier in HD patients?

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.

Dr Rachel HardingAugust 04, 2021

A first for CRISPR gene editing could have wider applications for human disease

A first for CRISPR gene editing could have wider applications for human 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?

Daniel O’ReillyJuly 21, 2021

Scientists identify precisely how pridopidine works in models of Huntington’s disease

Scientists identify precisely how pridopidine works in models of Huntington’s disease

New studies pinpoint precisely how the drug pridopidine works in models of Huntington’s disease

Dr Rachel HardingJune 12, 2021

Huntington's disease therapeutics conference 2021 - Day 3

Huntington's disease therapeutics conference 2021 - Day 3

Catch up on all of the latest updates from day 3 of the 2021 CHDI Huntington’s disease therapeutics conference #HDTC2021

Dr Rachel Harding, Dr Leora Fox, and Professor Ed WildApril 29, 2021

Huntington’s disease therapeutics conference 2021 - Day 1

Huntington’s disease therapeutics conference 2021 - Day 1

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.

Dr Rachel Harding, Dr Leora Fox, and Dr Jeff CarrollApril 28, 2021

Huntington's disease therapeutics conference 2021 - Day 2

Huntington's disease therapeutics conference 2021 - Day 2

Catch up on all of the latest updates from day 2 of the 2021 CHDI Huntington’s disease therapeutics conference #HDTC2021

Dr Rachel Harding, Dr Leora Fox, Dr Jeff Carroll, and Professor Ed WildApril 28, 2021

Huntington’s disease clinical trial round up

Huntington’s disease clinical trial round up

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

Dr Rachel HardingApril 26, 2021

Good news from uniQure: gene therapy trial on track, and promising data in animals

Good news from uniQure: gene therapy trial on track, and promising data in animals

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.

Dr Leora FoxApril 15, 2021

Disappointing Results from Wave’s PRECISION-HD1 and 2 Trials

Disappointing Results from Wave’s PRECISION-HD1 and 2 Trials

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.

Dr Rachel Harding and Dr Leora FoxMarch 30, 2021