secwatch.observer — SEC 8-K summary
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Issuer:     uniQure N.V. (QURE)
CIK:        0001590560
Form:       8-K
Filed at:   2024-08-15T23:59:59+00:00
Accession:  0001558370-24-012295
Event type: other_material
Sentiment:  neutral
Materiality: 0.55
Item codes: 7.01, 9.01
LLM model:  deepseek-v4-flash:cloud@v2

uniQure doses first patient in Phase I/IIa AMT-191 Fabry disease trial
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- First patient dosed in open-label, two-cohort Phase I/IIa trial of AMT-191 gene therapy for Fabry disease.
- Low-dose cohort: 6e10^13 gc/kg; high-dose: 3e10^14 gc/kg; one-time IV infusion; 24-month follow-up.
- AMT-191 uses same AAV5 delivery as HEMGENIX; targets liver to produce GLA protein.
- CEO cites catalysts: FDA engagement for AMT-130 (Huntington's), new trials in epilepsy and SOD1-ALS.
- Cash runway extended through end of 2027 from cost reduction initiatives.

Source:
  EDGAR index:    https://www.sec.gov/Archives/edgar/data/1590560/000155837024012295/0001558370-24-012295-index.htm
  Primary doc:    https://www.sec.gov/Archives/edgar/data/1590560/000155837024012295/qure-20240815x8k.htm
  HTML page:      https://secwatch.observer/filing/0001558370-24-012295

License: Source filings: public domain (SEC EDGAR). Summaries (headline + bullets): CC-BY-4.0; attribute https://secwatch.observer