Events & Presentations

Investor Presentations

• Virtual Research & Development Event – Nov. 29 event focused on preclinical gene therapy candidate for refractory temporal lobe epilepsy (rTLE) AMT-260 and uniQure AAV manufacturing advancements

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• AMT-130 12-Month Low-Dose Cohort Update – Investor Webcast Presentation

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• ESGCT 2022 – Enhancing efficacy and specificity of AAV5 capsid via small, intermediate, and large peptide insertions

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• EAHAD 2022 – Final Analysis from the Pivotal Phase 3 HOPE-B Gene Therapy Trial: Stable Steady-State Efficacy and Safety of Etranacogene Dezaparvovec in Adults with Severe or Moderately Severe Hemophilia B

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• ESGCT 2022 – linQURE™ platform: Development of novel scaffolds for multi-transcript targeting towards the treatment of complex genetic disorders

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• ISTH 2021 – Clinical Outcomes in Adults with Hemophilia B With Pre-existing Neutralizing Antibodies to AAV5: 6 Month Data from the HOPE-B Trial

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• ESGCT 2022 – AAV-miQURE®-mediated targeting of hexanucleotide repeat expansion-containing transcripts in ALS C9orf72 mouse models

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• Gene Therapy Information

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• ESGCT 2022 – Overexpressing a protective variant while simultaneously lowering toxic APOE as potential treatment for Alzheimer Disease

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• ESGCT 2022 – Development of a novel AAV9 gene therapy for the treatment of temporal lobe epilepsy using animal model and human organotypic slices

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• ESGCT 2022 – Biodistribution and safety of a novel AAV9 gene therapy for treatment of temporal lobe epilepsy shown in non-human primates

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• ESGCT 2022 – Scale-down of AAV production from commercial scale to a high-throughput mini-bioreactor system

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• ESGCT 2022 – Impact of bioreactor control parameters on AAV production

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