uniQure Announces Significant Presence at Upcoming American Society of Gene and Cell Therapy (ASGCT) Virtual Annual Meeting
April 28 2020 - 4:51PM
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, today announced that 22 data presentations, of which five
are oral presentations, will be delivered at the American Society
of Gene and Cell Therapy (ASGCT) Virtual 2020 Annual Meeting being
held May 12-15.
"uniQure’s significant scientific presence at
this year’s ASGCT Annual Meeting demonstrates the robustness of our
research capabilities and our excellence in developing novel
technologies and commercial-scale gene therapy manufacturing,”
stated Sander van Deventer, executive vice president of research
and product development at uniQure. “We are very pleased to present
new preclinical data on our gene therapy candidates for hemophilia
A, spinocerebellar ataxia type 3, Fabry disease and data on our
advances in technology and manufacturing.”
Specific details on uniQure’s virtual oral
presentations at ASGCT include:
Title: One-Time Intrathecal
Administration of AAV5-miATXN3 in Non-Human PrimatesDate
and Time: Wednesday May 13, 4:15 p.m. EDT/ 10:15 p.m.
CET
Title: A Novel NAGA Variant
Designed to be Non-immunogenic In Humans and Provide Broad
Cross-Correction in Fabry DiseaseDate and Time:
Thursday May 14, 4:15 p.m. EDT/ 10:15 p.m. CET
Title: A Single Administration
of AAV5-hFIX in Newborn, Juvenile and Adult Mice Leads to Stable
hFIX Expression up to 18 Months after DosingDate and
Time: Thursday May 14, 4:15 p.m. EDT/ 10:15 p.m. CET
Title: Characterizing
Next-Generation Baculovirus Transduction Processes - A Quality by
Design-based Approach for AAV ManufacturingDate and
Time: Friday May 15, 10:45 a.m. EDT/ 4:45 p.m. CET
Title: Clearance of Vector DNA
From Bodily Fluids in Patients with Severe or Moderate-Severe
Hemophilia B Following Systemic Administration of AAV5-hFIX and
AAV5-hFIX PaduaDate and Time: Friday May 15, 11:15
a.m. EDT/ 5:15 p.m. CET
The following presentations were approved for
poster presentation:
- Lipid Nanoparticle Pre-Treatment Improves rAAV Diffusion in the
Primate Liver and Enables an Increase of Therapeutic Transgene
Expression
- Prevalence and Avidity Assessment of Pre-existing Neutralizing
Antibodies (NABs) Against Adeno-Associated Virus (AAV) Vector
Serotypes 2, 5 And 8 Analyzed in The Serum Of 300 Healthy
Donors
- AAV Biology Imaging Platform: Uncovering the Cellular
Mechanisms Behind the AAV5-Based Vector Delivery System
- Development and Optimization of a qPCR Method to Assess
Biodistribution of an AAV5 Vector In NHP And Mouse
Studies
- Translatable Biomarkers in Gene Therapy for Huntington Disease:
Innovative Approaches and Learnings from Pre-Clinic to the
Clinic
- Exploring the Effects of Intrastriatal AAV5-miHTT Lowering
Therapy on Transcriptional Dysregulation, MRS Signal, and Mutant
Huntingtin Levels in the Q175FDN Mouse Model of Huntington’s
Disease
- Secreted Therapeutics: Monitoring Durability of microRNA-based
Gene Therapies in Huntington’s Disease
- Lowering the Pathogenic Exon 1 HTT Fragment by AAV5-miRNA Gene
Therapy
- Human Dose Prediction of a Novel Factor IX Variant Gene Therapy
Candidate (AMT-180) Mediating Clotting Independently of Factor
VIII
- Assessment of miQURE™ Efficacy and Safety in SCA3
Neurons
- Assessment of the Novel AAV-Based miQURE™ Gene Therapy in SCA3
Animal Models
- Development of an AAV5-Based Gene Therapy for
Dyslipidemia
- Generation and Optimization of Insect Based Stable AAV
Production Cell-Line
- Generation of a DuoBac Expression System for Robust and High
Quality AAV Production
- From Rocking Motion Bioreactors to Stirred Tank
Bioreactors the Journey to Scalable AAV Gene Therapy
- Production of Recombinant AAV Vectors in Chemically Defined
Media
- Overcoming Challenges for Developing AAV Purification Process
for Large-Scale GMP Manufacturing
The abstracts were published today at the ASGCT
Annual Meeting website.
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, hemophilia A, Huntington's disease,
Fabry disease, spinocerebellar ataxia Type 3 and other
diseases. www.uniQure.com
uniQure Contacts: |
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FOR
INVESTORS |
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FOR
MEDIA: |
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Maria E. Cantor |
Eva
M. Mulder |
Tom
Malone |
Direct:
339-970-7536 |
Direct: +31
20 240 6103 |
Direct:
339-970-7558 |
Mobile:
617-680-9452 |
Mobile: +31
6 52 33 15 79 |
Mobile:339-223-8541 |
m.canto@uniQure.com |
e.mulder@uniQure.com |
t.malone@uniQure.com |
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