gene-therapy
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Interruptions are encouraged
A CRISPR-based approach called ābase editingā is being explored to develop a new potential treatment for Huntington's disease. Editing a single letter in the genetic code with base editing may be the key for delaying HD symptoms, maybe by a decade.
By Dr Chris Kay -
Buckle in: Gene therapy AMT-130 appears to slow down signs of Huntingtonās 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
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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 itās paving the way for similar drugs targeting other diseases. Is Huntingtonās disease next?
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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
By Dr Leora Fox -
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.
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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.
By Dr Leora Fox -
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 uniQureās gene therapy for Huntingtonās 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.
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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. Letās talk more about what it means.
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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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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?