Two new participants have enrolled in gene therapy trial

In June, two people enrolled in the first gene therapy trial for Huntington’s disease. Now, two more participants have joined the study uniQure states in a new press release. 

UniQure is currently developing a gene therapy for Huntington’s disease. The drug candidate is called AMT-130 and is a gene therapy. In other words, it seeks to permanently change the genetic makeup.

This permanent change is the reason why AMT-130 only have to be administered one time: the effect of the drug is expected to last for a very long time, potentially lifelong.

AMT-130 is given through an injection directly into the brain. Of the first two participants enrolled this summer, one received AMT-130 – another an imitation surgery. 

The imitation surgery is an important part of the trial because it helps researchers to isolate the actual effects of the treatment – in this case AMT-130 – as opposed to potential side effects from undergoing surgery (such as pre- and postoperative care, the patient’s perception of having had a regular operation, etc.).

Learn how the drug candidate AMT-130 is administered:

The delivery method may sound scary but multiple studies have shown it to be highly precise and safe. Moreover, it will only be a one-time treatment. 

No significant safety concerns

The 90-day follow-up data from the first two participants indicate that the drug candidate is safe. Two more participants were therefore enrolled this month.

Early next year, a new safety evaluation will be carried out. If the results still show no significant safety concerns regarding AMT-130, enrollment of the first 10 participants is expected to be completed in mid-2021.

In total, 26 people with early manifest Huntington’s disease will participate in uniQure’s gene therapy trial. 

In a recently issued press release, Ricardo Dolmetsch, Ph.D., president of research and development at uniQure, stated: 

– This is an important achievement that puts us on our original clinical development timeline, making up for the modest delay in the study earlier this year due to COVID-19. 

Over the next years, uniQure will study how safe, tolerated and effective AMT-130 is.  

How does it work?

AMT-130 consists of a harmless virus – known as an adeno-associated virus (AAV) – which deliver instructions to neurons. The neurons are then told to make their own huntingtin lowering drug.

So while the person will still carry the disease-causing Huntington gene, the gene will work somewhat different and produce less huntingtin protein.

Learn more: Details emerge of first Huntington’s disease gene therapy clinical trial

How does this impact Huntington’s disease? By lowering the level of huntingtin protein – the harmful protein responsible for Huntington’s disease – uniQure is perhaps able to slow down, or even stop, the progression of the disease.

Learn more about how adeno-associated virus gene transfer works: