Articles with the topic: huntingtin-lowering

Real talk: Q&A with Roche about GENERATION-HD1

Real talk: Q&A with Roche about GENERATION-HD1

The HDBuzz team sat down (virtually) for an in depth Q&A session with the team at Roche to answer questions about tominersen and the recent halting of the GENERATION-HD1 trial

Dr Sarah Hernandez and Dr Leora FoxSeptember 28, 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

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

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

GPR52: Exploring a new way to lower huntingtin

GPR52: Exploring a new way to lower huntingtin

A Chinese research team developed a new way to lower huntingtin protein indirectly, by targeting a protein called GPR52. The molecules they designed were protective in cells and in mice with HD.

Dr Rachel HardingFebruary 11, 2021

Treatment for neurological disorder could be repurposed for Huntington’s disease patients

Treatment for neurological disorder could be repurposed for Huntington’s disease patients

While developing a drug called branaplam for patients with SMA, the pharmaceutical company Novartis discovered that it could hold promise for people with HD. The FDA has granted a special status called Orphan Drug Designation.

Dr Rachel HardingOctober 22, 2020