uniQure Announces Multiple Presentations at Upcoming American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

LEXINGTON, Mass. and AMSTERDAM, May 02, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that six data presentations, including one oral presentation, will be delivered at the American Society of Gene and Cell Therapy (ASGCT) Hybrid Congress 2022 Annual Meeting being held May 16-19 in Washington, D.C.

"Our presentations at ASGCT showcase the exceptional work of our uniQure scientists and the breadth of our research pipeline as we advance our investigational AAV gene therapy programs,” stated Ricardo Dolmetsch, Ph.D. president of research and development at uniQure. "We are particularly proud of our studies demonstrating the potential of the linQURE® cargo platform, delivering multiple miRNAs in a single AAV and its application to selectively silence mutant C9orf72 in an ALS preclinical model.”

Specific details on uniQure’s presentations taking place at ASGCT include:

• Title: Alpha-synuclein lowering and rescue of motor phenotype by miRNA-based AAV gene therapy in in vivo Parkinson’s disease models (Abstract #19)
  Presenter: Astrid Valles-Sanchez, Ph.D. director of CNS disease biology, uniQure
  Oral Session Title: Oligonucleotide Therapeutics
  Date and Time: Monday, May 16, 11:15 – 11:30 a.m. EDT

• Title: Construction and optimization of novel adeno-associated virus (AAV) rep gene expression control system for stable rAAV-packaging insect cell line (Abstract #407)
  Poster Session Title: Vector Product Engineering, Development or Manufacturing I
  Session Date and Time: Monday, May 16, 5:30 – 6:30 p.m. EDT

• Title: Functional transfer of engineered microRNAs through extracellular vesicles: An in vitro proof-of-mechanism study in Huntington’s disease models (Abstract #590)
  Poster Session Title: Oligonucleotide Therapeutics II
  Session Date and Time: Tuesday, May 17, 5:30 – 6:30 p.m. EDT

• Title: Selective silencing of mutant C9orf72 transcripts (Abstract #552)
  Poster Session Title: Gene Targeting and Gene Correction II
  Date and Time: Tuesday, May 17, 5:30 – 6:30 p.m. EDT

• Title: AAV5-GLA gene therapy results in sustained long term GLA transgene expression and cross correction of target organs in Fabry disease mouse model (Abstract #1009)
  Poster Session Title: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases II
  Session Date and Time: Wednesday, May 18, 5:30 p.m. EDT

• Title: AAV-mediated APOE gene silencing for Alzheimer’s disease (Abstract #918)
  Poster Session Title: AAV Vectors - Preclinical and Proof-of-concept Studies III
  Date and Time: Wednesday, May 18, 5:30 p.m. EDT

About uniQure
uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, refractory temporal lobe epilepsy, Fabry disease, and other diseases. www.uniQure.com

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