Articles with the topic: drug-development

New molecule can reverse the Huntington's disease mutation in lab models

New molecule can reverse the Huntington's disease mutation in lab models

A collaborative team of scientists from Canada and Japan have identified a small molecule which can change the CAG-repeat length in different lab models of Huntington's disease. #HuntingtonsDisease #DrugDiscovery

Dr Michael FlowerApril 06, 2020

What does COVID-19 mean for Huntington’s disease families and HD research?

What does COVID-19 mean for Huntington’s disease families and HD research?

COVID-19 update: what does it mean for HD families, how does it impact HD research, and how has it changed the way science works?

Dr Sarah Hernandez and Dr Ed WildApril 06, 2020

Huntington’s disease therapeutics conference 2020 - Day 1

Huntington’s disease therapeutics conference 2020 - Day 1

HDBuzz reports from the annual Huntington’s disease therapeutics conference in Palm Springs

Dr Rachel Harding, Dr Sarah Hernandez, and Joel StantonMarch 12, 2020

Unpacking Wave's PRECISION-HD2 huntingtin-lowering trial announcement

Unpacking Wave's PRECISION-HD2 huntingtin-lowering trial announcement

Wave Life Sciences announces that its antisense drug WVE-120102 has lowered mutant huntingtin protein in cerebrospinal fluid, but investors seem disappointed. Rather confusing – what do we know for sure?

Dr Jeff CarrollJanuary 03, 2020

Could molecular handcuffs lower the protein that causes Huntington's disease?

Could molecular handcuffs lower the protein that causes Huntington's disease?

Researchers got surprisingly lucky when looking for drug molecules to pull mutant huntingtin protein into a cellular garbage disposal machine

Dr Tamara MaiuriNovember 12, 2019

Exciting new Huntingtin lowering tool described

Exciting new Huntingtin lowering tool described

Exciting new Huntingtin Lowering work from @SangamoTx and @CHDIfoundation using "Zinc Fingers" to shut down expression of the mutant Huntingtin gene. More details on this exciting new technique here.

Dr Jeff CarrollAugust 05, 2019

Fresh Updates from First Huntingtin Lowering Study Publication

Fresh Updates from First Huntingtin Lowering Study Publication

Hot off the presses - New publication gives more details about the results of Ionis and Roche's safety study with a Huntingtin-lowering ASO

Dr Jeff CarrollMay 07, 2019

Huntington's disease goes viral as UniQure inches ahead in gene therapy race

Huntington's disease goes viral as UniQure inches ahead in gene therapy race

FDA grants “Investigational New Drug” status to Huntingtin-lowering gene-therapy agent AMT-130, clearing path to human trials in Huntington’s Disease patients

Dr Jeff CarrollJanuary 30, 2019

Advances on many fronts in the battle against the protein that causes Huntington's disease

Advances on many fronts in the battle against the protein that causes Huntington's disease

This fall sees exciting announcements from a number of companies focused on novel Huntingtin Lowering technologies, including Wave, PTC and Voyager

Dr Jeff CarrollDecember 04, 2018