# uniQure Huntington’s disease gene therapy regulatory filings

> Live situation record from CLSTR: https://clstr.news/situations/uniqure-huntingtons-disease-gene-therapy-regulatory-filings
> Updated: 2026-10-03T20:17:48.000Z. Sources: 9. Developments: 3.

uniQure has initiated regulatory approval processes for AMT-130, a gene therapy designed to treat the underlying cause of Huntington’s disease. The company submitted a Biologics License Application to the U.S. Food and Drug Administration (FDA) requesting accelerated approval and a priority review, while also filing a marketing application with the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA).

Updated Phase I/II clinical data through the 4-year mark indicates the therapy continues to show a slowing of disease progression, though recent results are mixed. While 36-month data showed high-dose patients demonstrated an 80% slowing of decline on the Composite Unified Huntington’s Disease Rating Scale (cUHDRS) and a 67% slowing on Total Functional Capacity (TFC), the 48-month analysis of 12 high-dose patients showed a 44% slowing of decline on the cUHDRS that did not reach statistical significance (p-value of 0.144). However, the TFC metric showed a 61% slowing at 48 months.

Following the validation of its marketing authorization application by the MHRA, uniQure expects an acceptance decision from the FDA during the fourth quarter and plans to begin screening patients for a confirmatory study before the end of the year. Despite the nuanced long-term data, Barclays has maintained a buy recommendation for the company. Financially, uniQure reported a revenue increase to 5.84 million USD in the second quarter of 2026, while its GAAP net loss expanded to 81.06 million USD.

## Timeline

### 2026-10-03: uniQure releases mixed 48-month clinical data for AMT-130

uniQure's 48-month AMT-130 clinical data shows mixed results, with a 44% slowing on the cUHDRS scale failing to reach statistical significance, prompting financial re-evaluations.

3 sources. https://clstr.news/cluster/uniqure-releases-mixed-48-month-clinical-data-for-amt-130

### 2026-09-29: uniQure reports 4-year data for Huntington’s disease gene therapy AMT-130

uniQure reported updated Phase I/II data for its Huntington’s disease gene therapy, AMT-130, showing continued disease progression slowing at 48 months despite some 48-month metrics lacking statistical weight.

5 sources. https://clstr.news/cluster/uniqure-files-fda-application-for-huntingtons-disease-gene-therapy

### 2026-09-02: uniQure seeks U.S. and U.K. approval for Huntington's gene therapy

uniQure has applied for accelerated approval of its gene therapy, AMT-130, for Huntington’s disease in the U.S. and U.K., seeking to provide the first treatment that targets the disease's underlying cause.

2 sources. https://clstr.news/cluster/uniqure-seeks-us-and-uk-approval-for-huntingtons-gene-therapy

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Cite as: uniQure Huntington’s disease gene therapy regulatory filings. CLSTR, https://clstr.news/situations/uniqure-huntingtons-disease-gene-therapy-regulatory-filings