$QURE (+0,31 %)

uniQure schedules FDA meeting to discuss accelerated approval of HD therapy

🔹 Key message

uniQure is planning an official discussion with the US Food and Drug Administration (FDA) to accelerated approval for its gene therapy AMT-130 for the treatment of Huntington's disease for the treatment of Huntington's disease.

🔍 Details

• The company is preparing for a pre-BLA meeting with the FDA (BLA = Biologics License Application).
• The aim is to bring the therapy bring the therapy to market more quicklyas Huntington's disease is a a rare, fatal neurodegenerative disease. disease.
• uniQure sees positive clinical data as the basis for the application for accelerated approval

📈 Market impact

• The news has caused uniQure's share price to rise significantly.
• Investors see this as a potential turning point for the company.

🧠 Background to AMT-130

• AMT-130 is a unique gene therapythat targets the cause of Huntington's disease.
• The therapy utilizes AAV vectorsto deliver a gene into the central nervous system.

$QURE (+0,31 %)

A breakthrough in a previously incurable disease catapults the uniQure share to unimagined heights. The gene therapy AMT-130 shows convincing efficacy against Huntington's disease for the first time.

Share price jumps after medical milestone

- Breakthrough in Huntington's disease

- Study data show clear signs of efficacy

The uniQure share is experiencing a spectacular surge that is rare even in the volatile biotech sector. This was triggered by groundbreaking results from the Phase I/II trial of the gene therapy AMT-130, which significantly slowed down the progression of the disease in Huntington's disease patients. The scientific success catapults the share price in Frankfurt temporarily up 266.74 percent to 42.23 euros.

What is particularly remarkable is that the therapy promises a long-term effect with a single treatment for a disease for which there are hardly any treatment options to date. For the first time, the study data show clear signs of efficacy with an acceptable safety profile - a breakthrough that gives hope to Huntington's disease patients worldwide.

Billion-dollar market in sight - but still hurdles to overcome

For investors, uniQure could be at the beginning of a success story. What was previously just a research project in the pipeline is now developing into a potential blockbuster product with sales in the billions. The company is already preparing itself financially for the approval process and a possible market launch.

Nevertheless, despite the euphoria, the share remains a speculative investment. The decisive Phase III trial is still pending, and only the results of this will determine whether the product is actually eligible for approval.

What makes the AMT-130 gene therapy so revolutionary

Huntington's disease is a hereditary neurodegenerative disease that is currently considered incurable. AMT-130 could change this by tackling the root of the problem directly with gene therapy. In contrast to conventional drugs, which only alleviate symptoms, this treatment promises to fundamentally change the course of the disease.

If the therapy successfully passes all further study phases, this would not only represent a breakthrough for Huntington's disease patients, but could also boost research into other gene therapies. uniQure is thus positioning itself as a pioneer in a promising segment of biotechnology.

finanzen.net editorial team

https://www.finanzen.net/nachricht/aktien/hoehenflug-uniqure-aktie-schiesst-266-prozent-hoch-huntington-erfolg-treibt-an-00-14886817