Articles with the topic: drug-development

Artificial intelligence to save the day? How clever computers are helping us understand Huntington’s disease.

Artificial intelligence to save the day? How clever computers are helping us understand Huntington’s disease.

Scientists at IBM and the CHDI Foundation have used artificial intelligence to analyse datasets from Huntington’s disease observational trials to model progression of the disease. They hope their findings will help improve clinical trial design.

Dr Rachel HardingJune 21, 2022

A spoonful of branaplam helps the huntingtin go down

A spoonful of branaplam helps the huntingtin go down

Branaplam was originally designed to treat spinal muscular atrophy, but a new paper outlines how it could hold promise for treating Huntington’s. This oral drug lowers huntingtin protein and will now be tested in a study called VIBRANT-HD.

Dr Rachel HardingMarch 16, 2022

Oral drug may change the story for huntingtin lowering

Oral drug may change the story for huntingtin lowering

Researchers with PTC Therapeutics recently published exciting new findings - a promising new huntingtin lowering drug that can be taken as a pill. Will this change how we move forward with huntingtin lowering?

Dr Sarah Hernandez and Dr Jeff CarrollFebruary 01, 2022

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

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

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

Sad News from Roche and Ionis - ASO Trial Halted Early

Sad News from Roche and Ionis - ASO Trial Halted Early

Disappointing news from Roche and Ionis; the phase III Tominersen huntingtin-lowering trial has been halted early

Dr Jeff Carroll and Dr Rachel HardingMarch 23, 2021

Uncovering the dark side of DNA repair to design HD treatments

Uncovering the dark side of DNA repair to design HD treatments

A gene called MSH3 helps to repair our DNA, but in HD it can slip up and cause CAG repeats to lengthen. Researchers have uncovered new information about how MSH3 activity is controlled, opening the door to new therapeutic avenues.

Emma BuntingDecember 22, 2020