secwatch.observer — SEC 8-K summary
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Issuer:     uniQure N.V. (QURE)
CIK:        0001590560
Form:       8-K
Filed at:   2024-10-15T23:59:59+00:00
Accession:  0001558370-24-013276
Event type: other_material
Sentiment:  neutral
Materiality: 0.30
Item codes: 7.01, 9.01
LLM model:  deepseek-v4-flash:cloud@v2

uniQure doses first patient in Phase I/II trial of AMT-162 gene therapy for SOD1-ALS
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- First patient dosed in EPISOD1 Phase I/II open-label trial of AMT-162 for SOD1-ALS, a rare inherited progressive motor neuron disease.
- Trial has three dose-escalating cohorts; up to four patients per cohort receiving intrathecal AMT-162 with immunosuppression.
- AMT-162 is an AAVrh10-based gene therapy expressing miRNA to knock down mutated SOD1 protein; granted Orphan Drug and Fast Track FDA designations.
- Four active U.S. sites; plans to activate seven additional sites by Q1 2025.
- Trial will assess safety, tolerability, and exploratory efficacy via neurofilament light chain and SOD1 protein biomarkers.

Source:
  EDGAR index:    https://www.sec.gov/Archives/edgar/data/1590560/000155837024013276/0001558370-24-013276-index.htm
  Primary doc:    https://www.sec.gov/Archives/edgar/data/1590560/000155837024013276/qure-20241015x8k.htm
  HTML page:      https://secwatch.observer/filing/0001558370-24-013276

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