A groundbreaking clinical trial by uniQure has demonstrated that their gene therapy, AMT-130, can slow Huntington's disease progression by 75 percent over three years, marking a major milestone in neurodegenerative disease treatment.

The therapy involves a delicate brain surgery where a modified virus delivers DNA to reduce levels of the toxic huntingtin protein, which is responsible for neuron death, offering hope for extending patients' quality of life.

While promising, the treatment is not a cure and involves complex, lengthy brain surgery, with safety profiles showing manageable adverse events mainly related to the procedure, which have resolved without major issues.

The trial results suggest that early intervention could help at-risk individuals, potentially delaying or halting disease progression, and some patients have shown remarkable improvements, including returning to work and maintaining mobility longer than expected.

The positive data has led to a surge in uniQure's stock, which soared approximately 190%, reaching a new 52-week high of $39.59, reflecting investor optimism and validation of years of development.

Analysts are overwhelmingly bullish on the stock, with nine out of eleven rating it a Buy or Strong Buy, and price targets averaging between $37 and $40, some raised following the positive trial results.

The therapy was generally well-tolerated, with most adverse events related to anesthesia and surgery that resolved without major issues, and some patients experienced stability or improvement, including returning to work.

Regulatory approval in the US is scheduled for early 2026, with plans to seek approval in Europe and the UK later, though the high complexity and costs of the procedure may limit accessibility.

UniQure remains in early commercialization, reporting modest revenues mainly from royalties, with a cash runway into late 2027 supported by significant cash reserves and a new $175 million non-dilutive loan to support the therapy's launch.

Despite the promising results, the Huntington's field remains challenging, with other companies experiencing setbacks, but the success of AMT-130 supports the broader potential of gene therapy for neurodegenerative diseases.

The breakthrough could encourage more at-risk individuals to undergo genetic testing, as treatment options now exist, potentially transforming the landscape of Huntington's disease management.