Articles with the topic: gene-therapy

Buckle in: Gene therapy AMT-130 appears to slow down signs of Huntington’s disease in Phase I/II clinical trial

Buckle in: Gene therapy AMT-130 appears to slow down signs of Huntington’s disease in Phase I/II clinical trial

More good news for the HD community from uniQure, whose one-and-done gene therapy appears safe and shows hints of slowing down signs and symptoms of the disease

Dr Rachel HardingAugust 16, 2024

Steady progress from uniQure - promising data to end the year

Steady progress from uniQure - promising data to end the year

uniQure ushered in the end of the year by releasing some promising data from their huntingtin-lowering gene therapy trials

Dr Leora FoxAugust 16, 2024

CRISPR-based drugs: one giant leap for mankind

CRISPR-based drugs: one giant leap for mankind

Casgevy is the first CRISPR-based drug to make its way through the approval process, all but curing Sickle Cell Disease and it’s paving the way for similar drugs targeting other diseases. Is Huntington’s disease next?

Dr Sarah HernandezFebruary 14, 2024

Updates from PTC Therapeutics and uniQure on their huntingtin-lowering trials

Updates from PTC Therapeutics and uniQure on their huntingtin-lowering trials

Both PTC Therapeutics and uniQure have shared updates from their respective clinical trials, testing different huntingtin-lowering approaches. We explore the data presented from both of these studies and what this means for HD family members.

Dr Leora Fox and Dr Rachel HardingJune 21, 2023

uniQure gets the green light to resume testing HD gene therapy

uniQure gets the green light to resume testing HD gene therapy

Following a 3-month pause in enrollment due to concerns about side effects, uniQure shared the good news that their trial of the HD gene therapy AMT-130 will continue as planned, with new safety measures in place.

Dr Leora FoxNovember 03, 2022

Serious side effects reported for some people treated with the huntingtin-lowering drug AMT-130, currently in clinical trials

Serious side effects reported for some people treated with the huntingtin-lowering drug AMT-130, currently in clinical trials

After receiving a high dose of uniQure’s gene therapy for Huntington’s disease, a few patients experienced serious side effects, but are now recovering. HDBuzz explores what this means for the experimental huntingtin-lowering drug, AMT-130.

Dr Sarah Hernandez and Dr Rachel HardingAugust 29, 2022

Updates from uniQure about their gene therapy for Huntington's disease

Updates from uniQure about their gene therapy for Huntington's disease

uniQure is conducting safety trials of the first gene therapy for HD. A recent press release provided a 1 year update on the first group of 10 people who underwent surgery to receive this experimental drug. Let’s talk more about what it means.

Dr Leora Fox and Dr Rachel HardingJuly 11, 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

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