Lexington, Massachusetts-based uniQure N.V. (Nasdaq: QURE) announced a proposed underwritten public offering of ordinary shares and pre-funded warrants targeting USD 150 million in gross proceeds, as the gene therapy company advances toward a planned Biologics License Application (BLA) submission for its lead Huntington's disease candidate, AMT-130. The financing comes shortly after the company disclosed that the US FDA had indicated three-year Phase I/II data could support an accelerated approval filing, materially improving visibility on the program's regulatory pathway.
The offering consists of ordinary shares and, in lieu of ordinary shares to certain investors, pre-funded warrants to purchase ordinary shares. uniQure has also granted underwriters a 30-day option to purchase up to an additional USD 22.5 million of ordinary shares, bringing potential gross proceeds to USD 172.5 million. Leerink Partners and Stifel are acting as joint bookrunning managers. Pricing, share count, and final use of proceeds were not disclosed.
Company overview and pipeline
uniQure is a gene therapy company focused on neurological and rare genetic diseases. Its approved product, hemophilia B gene therapy Hemgenix (etranacogene dezaparvovec), is commercialized by CSL Behring under a licensing agreement.
The company's lead wholly owned asset is AMT-130, an investigational AAV5-based gene therapy for Huntington's disease. In June 2026, uniQure announced plans to submit a BLA in Q3 2026 after the FDA indicated that three-year Phase I/II data could serve as the primary basis for an accelerated approval application. High-dose AMT-130 demonstrated a statistically significant 75% slowing of disease progression versus a propensity score-matched external control cohort in three-year follow-up analyses.