Articles with the topic: drug-development

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

HDBuzz Enrolls in Enroll-HD

HDBuzz Enrolls in Enroll-HD

As promised, HDBuzz's Jeff Carroll finally signed up for the Enroll-HD study. Learn more about this important global HD "observational study" here.

Dr Jeff CarrollJuly 31, 2018

First Dolly, now Piglet; a new Huntington’s disease pig knock-in model confirmed

First Dolly, now Piglet; a new Huntington’s disease pig knock-in model confirmed

For the first time in HD research, an animal model has been created in pigs using 2 cutting edge DNA editing t

Caroline CaseyJuly 10, 2018

New collaboration seeks to speed Huntington's disease drug licensing

New collaboration seeks to speed Huntington's disease drug licensing

Critical Path Institute launches new initiative to get HD drugs licensed as quickly as possible

Dr Jeff CarrollMay 10, 2018

A new way of thinking about trials to prevent Huntington's disease

A new way of thinking about trials to prevent Huntington's disease

Can we test drugs to delay or prevent the onset of Huntington's disease? New research suggests it's possible

Dr Jeff CarrollOctober 27, 2017