uniQure Announces Significant Presence at Upcoming American Society of Gene and Cell Therapy (ASGCT) Virtual Annual Meeting - Seite 2

 

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

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