UniQure\u2019s experimental gene therapy for Huntington\u2019s disease slowed progression of the brain disorder by 75% in an early-to-mid stage study, it said on Wednesday.<\/p>\n
Shares of the company more than tripled in midday trading, skyrocketing 250%.<\/p>\n
Huntington\u2019s is a rare inherited brain disorder, which steadily worsens and typically leads to death 10 to 30 years after symptoms begin. There are no FDA-approved treatments for the condition.<\/p>\n
UniQure\u2019s therapy, called AMT-130, reduced disease progression by 75% at 36 months in patients who received a high dose, based on a widely used clinical scale.<\/p>\n
It also slowed decline of functional abilities in patients by 60%, a key secondary goal of the trial.
\n\u201cThese groundbreaking data are the most convincing in the field to date and underscore potential disease-modifying effects in Huntington\u2019s disease, where an urgent need persists,\u201d said Sarah Tabrizi, director of the University College London\u2019s Huntington\u2019s Disease Center.<\/p>\n
AMT-130 was generally well-tolerated, with no new serious side effects reported since late 2022, the company said.
\nUniQure plans to submit a marketing application to the U.S. Food and Drug Administration in early 2026, with hopes of launching the therapy later that year if approved.<\/p>\n
About 41,000 people in the U.S. are living with the disease, and more than 200,000 are at risk of inheriting it, according to Huntington\u2019s disease Society of America.<\/p>\n","protected":false},"excerpt":{"rendered":"
UniQure\u2019s experimental gene therapy for Huntington\u2019s disease slowed progression of the brain disorder by 75% in an early-to-mid stage study, it said on Wednesday. Shares of the company more than tripled in midday trading, skyrocketing 250%. Huntington\u2019s is a rare inherited brain disorder, which steadily worsens and typically leads to death 10 to 30 years […]<\/p>\n","protected":false},"author":2,"featured_media":1961,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[37],"tags":[559],"class_list":{"0":"post-1960","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-sitefansalaran-industry-news","8":"tag-uniqures-therapy-slows-huntingtons-disease-progression-in-trial-shares-more-than-triple"},"yoast_head":"\n