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UniQure Looks For Clear Signal From Huntington’s Disease Gene Therapy

Blockbuster Potential For AMT-130 In Neurodegenerative Disease

 
• By Andrew McConaghie

After recent landmark approvals in other neurodegenerative diseases such as Alzheimer’s and amyotrophic lateral sclerosis, hopes are rising that Huntington’s disease could be next to reach a breakthrough.

UniQure

UniQure is awaiting updated data from its potential first-in-class gene therapy, AMT-130, with the most important being a Phase I/II trial in the US monitoring a small group of patients at 12 and 24 months after treatment.

The gene therapy uses an AAV5 vector to transports a micro-RNA into the central nervous system with the goal of...

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