huntingtin-lowering
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Updates from the EHDN Meeting 2021
Last month, HDBuzz attended the online European Huntington's Disease Network (EHDN) meeting. Read our summary of all the latest clinical trial updates.
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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
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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.
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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.
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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
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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.
By Dr Leora Fox -
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.
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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
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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.
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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.