UniQure Raises $225 Million to Back Accelerated Approval Bid for Huntington’s Gene Therapy

Rare Daily Staff

UniQure is capitalizing on an improved regulatory outlook for its experimental Huntington’s disease gene therapy to price an upsized public offering of $225 million.

The company priced a little more than 4.9 million ordinary shares at $45.50 dollars per share to finance its accelerated approval push and planned confirmatory trial while bolstering its balance sheet and strategic flexibility.

UniQure has granted underwriters a 30‑day option to purchase up to 741,758 additional shares at the same public price, less underwriting fees. All shares are primary issuance by UniQure, and the offering is expected to close on or about June 25, 2026.

The offering follows a recent reversal by the U.S. Food and Drug Administration from its earlier stance that UniQure would need an additional study before it would consider approval of AMT‑130, the company’s Huntington’s disease gene therapy candidate. Instead, the agency has now agreed that mid‑stage phase 1/2 data could support an accelerated approval application.

For the planned application, UniQure intends to compare treated patients from early cohorts against a propensity score‑matched external control derived from the Enroll‑HD natural history dataset under a prespecified statistical analysis plan. Such external control strategies have become more common in rare disease development but remain subject to regulatory scrutiny, particularly in neurodegenerative indications.

Following a Type B meeting, FDA indicated that three‑year data from the ongoing phase 1/2 program may serve as the primary basis for the accelerated approval submission, provided there is alignment on the design of a confirmatory study. The agency expressed openness to a confirmatory trial using a concurrent standard‑of‑care control arm rather than a sham neurosurgical procedure, directly addressing ethical concerns around sham brain surgery in Huntington’s disease.

If accepted, the application would mark one of the first gene therapies targeting Huntington’s disease to reach the regulatory review stage and could further test FDA’s flexibility in applying accelerated approval pathways to neurodegenerative disorders with limited treatment options.

Huntington’s disease is a rare, inherited disorder caused by a CAG repeat expansion in the huntingtin gene, leading to progressive motor dysfunction, cognitive decline, and psychiatric symptoms. Approximately 75,000 people across the United States, Europe, and the United Kingdom are affected, with many more at risk of inheriting the condition.

AMT‑130 is an adeno‑associated virus‑based gene therapy delivered via one‑time, MRI‑guided stereotactic neurosurgical infusion into the striatum, designed to reduce production of the mutant huntingtin protein that drives Huntington’s disease pathology

AMT‑130 has Regenerative Medicine Advanced Therapy, Breakthrough Therapy, and Fast Track designations, underscoring the high unmet need and FDA’s willingness to apply flexible pathways in a rare neurodegenerative disorder where no approved therapies currently slow disease progression.

UniQure said it plans to file an application for accelerated approval in the third quarter of 2026.