uniQure N.V. (QURE): A Bull Case Theory 

We came across a bullish thesis on uniQure N.V. on Truffle Pigs’s Substack. In this article, we will summarize the bulls’ thesis on QURE. uniQure N.V.’s share was trading at $63.65 as of October 9th. QURE’s forward P/E was 27.93 according to Yahoo Finance.

uniQure’s AMT-130 represents a groundbreaking advancement in Huntington’s disease, providing the first credible evidence that a gene therapy can meaningfully alter the course of this fatal neurodegenerative disorder. Unlike previous therapies, AMT-130 achieved a 75% slowing of disease progression over three years in the high-dose cohort, with robust improvements across functional, cognitive, and motor measures as captured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS).

Key secondary endpoints, including Total Functional Capacity (TFC), showed a 60% reduction in functional decline, directly reflecting patients’ ability to live independently. Biomarker data, notably sustained declines in cerebrospinal-fluid neurofilament-light (NfL), further confirm neuroprotection and disease modification rather than transient symptomatic effects. Safety outcomes were manageable, with adverse events largely related to surgical delivery and resolving fully, supporting a favorable long-term profile.

Regulatory pathways for AMT-130 are highly favorable. uniQure plans a pre-BLA meeting with the FDA in Q4 2025, followed by a Biologics License Application (BLA) in early 2026, requesting Priority Review and likely leveraging the Commissioner’s National Priority Voucher (CNPV) program, which could accelerate approval to as little as two months.

Commercially, the therapy targets an initial U.S. patient population of roughly 6,000, with pricing around $2.5 million per treatment, implying a $15 billion market opportunity. The therapy’s transformative impact positions uniQure as an attractive takeover candidate for major pharmaceutical companies with neuroscience or gene therapy expertise, with historical acquisition multiples suggesting a potential $15–20 billion valuation.

AMT-130’s durable efficacy, clear regulatory path, substantial market opportunity, and potential for strategic acquisition create a uniquely compelling investment case. While development-stage biotech risks remain, including regulatory, clinical, and commercialization uncertainties, the therapy’s profile offers an exceptional risk/reward skew, establishing uniQure as a potential leader in Huntington’s disease and CNS gene therapies more broadly.

Previously we covered a bullish thesis on CRISPR Therapeutics AG (CRSP) by MADD-Scientis in March 2025, which highlighted the commercial potential of Casgevy, its breakthrough gene therapy for sickle cell disease, strong institutional backing, and a robust pipeline. The company’s stock price has appreciated approximately by 82.86% since our coverage, reflecting early adoption and regulatory progress. The thesis still stands as CRSP continues to advance its gene-editing platform. Truffle Pigs shares a similar focus but emphasizes uniQure’s AMT-130 for Huntington’s disease and its near-term FDA pathway.

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Disclosure: None.