uniQure updates gene therapy pipeline and regulatory outlook

The latest update is out from uniQure ( (QURE) ).

In January 2026, uniQure N.V. updated its corporate presentation to highlight its positioning as a leader in gene therapy, underscoring a robust clinical pipeline and substantial liquidity to fund development across multiple rare and serious neurological and metabolic diseases. The refreshed materials emphasize AMT-130 as a first potential disease-modifying, one-time gene therapy for Huntington’s disease with so-called blockbuster potential, outline upcoming data readouts for AMT-260 in mesial temporal lobe epilepsy and AMT-191 in Fabry disease over the next three to six months, and signal focused engagement with the U.S. Food and Drug Administration on a potential biologics license application pathway, developments that collectively underscore the company’s bid to consolidate its competitive position in gene therapy and may be closely watched by investors, patients and partners for signs of clinical and regulatory progress.

The most recent analyst rating on (QURE) stock is a Hold with a $22.00 price target. To see the full list of analyst forecasts on uniQure stock, see the QURE Stock Forecast page.

Spark’s Take on QURE Stock

According to Spark, TipRanks’ AI Analyst, QURE is a Neutral.

The score is held down primarily by weak financial performance (ongoing losses and negative cash flow) and a soft technical backdrop (below key moving averages with negative MACD). Offsetting factors include meaningful clinical progress and a strong cash position highlighted on the earnings call, but FDA/BLA uncertainty and rising expenses limit the upside in the overall rating.

To see Spark’s full report on QURE stock, click here.

More about uniQure

uniQure N.V. is a gene therapy pioneer with a 25-year history, built around an adeno-associated virus (AAV) platform and a commercial-ready manufacturing capability. The company focuses on developing one-time, potentially disease-modifying gene therapies, with an approved product in hemophilia B and a pipeline that includes AMT-130 for Huntington’s disease, AMT-260 for mesial temporal lobe epilepsy, AMT-191 for Fabry disease, and AMT-162 for SOD1-related amyotrophic lateral sclerosis. As of September 30, 2025, uniQure reported a strong financial position with approximately $694.2 million in cash, cash equivalents and investment securities, supporting its efforts to advance late- and early-stage programs and regulatory interactions, particularly in the U.S.

Average Trading Volume: 3,163,506

Technical Sentiment Signal: Buy

Current Market Cap: $1.56B

For an in-depth examination of QURE stock, go to TipRanks’ Overview page.