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uniQure Announces Major Presence at the European Society of Gene and Cell Therapy (ESGCT) Annual Meeting
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that 12 data presentations, including two oral presentations, will be delivered at the European Society of Gene and Cell Therapy (ESGCT) Annual Meeting being held October 24-27 in Brussels, Belgium.

“Our multiple presentations at ESGCT demonstrate uniQure’s continued leadership in advancing important research within the field of gene therapy, as well as highlight our industry-leading expertise in AAV gene therapy manufacturing,” stated Richard Porter, Ph.D., Chief Business and Scientific Officer at uniQure. “We look forward to sharing these presentations that feature our technology and platform experience across numerous programs.”

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

• Invited Talk Title: Update on U.S. Phase I/II Clinical Trial of AMT-130 Gene Therapy for the Treatment of Huntington’s DiseasePresenter: Richard Porter, Chief Business and Scientific Officer, uniQureSession Title and Location: CNS & sensory diseases II, Session 10a, Shed 2ADate and Time: Friday, October 27, 8:30 a.m. GMT+2

• Title: Gene therapy for C9ORF72-ALS reduces RNA toxicity and ameliorates behavioral phenotype in ALS mouse modelPresenter: Vanessa Zancanella, Sr. Scientist Global Research, uniQureSession Title and Location: CNS & sensory diseases I, Session 2c, Maison de la PosteDate and Time: Tuesday, October 24, 17:00 – 19:00 GMT+2

• Title: Pivotal safety studies for AMT-260, a novel AAV9-dual microRNA-based vector targeting GRIK2 for the treatment of temporal lobe epilepsy (Poster #433)Presenter: Nick Pearson, Sr. Director Toxicology & Translational Safety, Non-Clinical, uniQure Poster Session Date and Time: Wednesday, October 25, 17:00 – 18:15, Thursday, October 26, 20:30 – 21:30 GMT+2

• Title: Production of low cross-packaging AAV capsid libraries in insect cells (Poster #166)Presenter: Bas Bosma, Scientific Leader, Technology Innovation, uniQurePoster Session Date and Time: Wednesday, October 25, 18:15 – 19:30, Thursday October 26, 19:30 – 20:30 GMT+2

• Title: Multiplexed comparison of rationally-designed AAV CNS capsids in vivo (Poster #181)Presenter: Elena Pimentel, Scientist, Technology Innovation, uniQurePoster Session Date and Time: Wednesday, October 25, 17:00 – 18:15, Thursday, October 26, 20:30 – 21:30 GMT+2

• Title: Silence and replace as potential treatment for Alzheimer Disease: in concert lowering toxic APOE and augmenting protective APOE (Poster #049)Presenter: Amila Zuko, Scientist, Global Research, uniQurePoster Session Date and Time: Wednesday, October 25, 17:00 – 18:15, Thursday, October 26, 20:30 – 21:30 GMT+2

• Title: Gene therapy for synucleinopathies: Combining vectorized antibody and miRNA-mediated lowering strategies (Poster #430)Presenter: Kim Wolzak, Jr. Scientist, Global Research, uniQurePoster Session Date and Time: Wednesday, October 25, 18:15-19:30, Thursday, October 26, 19:30-20:30 GMT+2

• Title: Alpha-synuclein lowering and rescue of motor phenotype by miRNA-based AAV gene therapy in an in vivo Parkinson’s disease rat model (Poster #420)Presenter: Rhodé van Westen Erbrink, Scientist, Global Research, uniQurePoster Session Date and Time: Wednesday, October 25, 18:15-19:30, Thursday, October 26, 19:30-20:30 GMT+2

• Title: How uniQure’s modular platform approach drives continuous innovation in AAV gene therapy development and manufacturing (Poster #329)Presenter: Erich Ehlert, Director – Process Development – Chemistry, Manufacturing and Controls, uniQurePoster Session Date and Time: Wednesday, October 25, 17:00-18:15, Thursday, October 26, 20:30-21:30 GMT+2

• Title: Accelerating process development by establishing drug substance development platform (Poster #191)Presenter: Giorgio Rainone, Bioprocess Technologist, Drug Substance Development, uniQurePoster Session Date and Time: Wednesday, October 25, 17:00-18:15, Thursday, October 26, 20:30-21:30 GMT+2

• Title: High-throughput process development in rAAV drug substance manufacturing (Poster #308)Presenter: Yang Jiang, Associate Director, Drug Substance Development, uniQurePoster Session Date and Time: Wednesday, October 25, 18:15-19:30, Thursday, October 26, 19:30-20:30 GMT+2

• Title: Platform development for AAV manufacturing with the Baculovirus Expression Vector System (BEVS) (Poster #193)Presenter: Zandra Félix Garza, Project Manager- BPU – Cells, Seeds and Research Supply, uniQurePoster Session Date and Time: Wednesday, October 25, 17:00-18:15, Thursday, October 26, 20:30-21:30 GMT+2

About uniQure

uniQure’s mission is to reimagine the future of medicine by delivering innovative cures that transform lives. The recent approvals of our gene therapy for hemophilia B – an historic achievement based on more than a decade of research and clinical development – represent a major milestone in the field of genomic medicine and ushers in a new treatment approach for patients living with hemophilia. We are now leveraging our modular and validated technology and manufacturing platform to advance a pipeline of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory mesial temporal lobe epilepsy, amyotrophic lateral sclerosis (ALS), Fabry disease, and other severe diseases. www.uniQure.com

uniQure Contacts:

FOR INVESTORS:Chiara RussoDirect: 617-306-9137Mobile: 617-306-9137c.russo@uniQure.com

FOR MEDIA:Tom MaloneDirect: 339-970-7558Mobile:339-223-8541 t.malone@uniQure.com