Stock Monitor: Aevi Genomic Medicine Post Earnings Reporting

LONDON, UK / ACCESSWIRE / May 22, 2018 / If you want access to our free research report on uniQure N.V. (NASDAQ: QURE), all you need to do is sign up now by clicking the following link www.active-investors.com/registration-sg/?symbol=QURE as the Company's latest news hit the wire. On May 18, 2018, the Company announced that it has reported preclinical data in three poster presentations highlighting its expertise in efficient transduction of target cells within affected organs at American Society of Gene and Cell Therapy (ASGCT) 21st Annual Meeting held in Chicago, Illinois, May 17-18, 2018. The presentations highlighted that uniQure's modular platform is now broadly applicable to treat patients with both liver and central nervous system (CNS) disorders. Register today and get access to over 1,000 Free Research Reports by joining our site below:

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Highlights from Presentations at ASGCT

  • AAV5 Gene Delivery to the Central Nervous System: New data demonstrating transduction by AAV5 vectors in different cell types in the CNS, including astrocytes, motor neurons, and medium spiny neurons was presented. The data showed that AAV5 achieved a diffuse transduction profile via direct injection in the ventricles of the brain, suggesting that diseases with involvement of deep brain structures can be targeted with AAV5. Based on these data, AAV5 gene therapy may be applicable to a wide range of neurodegenerative diseases where broad coverage of the CNS is desired.
  • Increase of Therapeutic Transgene Expression in the Liver: Recent enhancements to the systemic administration of AAV5 gene therapies that substantially improve transduction in the liver was reported. First, a method that exactly determines the percentage of hepatocytes transduced following gene therapy in primates and simultaneously quantifies the expression of therapeutic protein for each transduced cell was described. Using this method, in a separate poster, the Company showed that pre-treatment with a lipid emulsion prior to AAV5 administration significantly improves the efficacy of AAV5 vector delivery to the liver and enables broader hepatic cell targeting throughout the tissue. By blocking the liver sinusoidal endocytotic capacity, pretreatment with a lipid emulsion significantly increased the percentage of transduced hepatocytes and caused a uniform distribution of the vector throughout the liver tissue. This approach, which can be readily implemented in future clinical trials, represents an important tool for future liver-targeted gene therapies.
  • uniQure also delivered an oral presentation examining the relationship between the presence of pre-existing anti-AAV5 neutralizing antibodies and clinical outcomes of AMT-060 in patients with hemophilia B.

uniQure Expertise Enables to Implement Novel Technologies that Lead to Transduction of a Target Cell Types in CNS and Liver

Sander van Deventer, M.D., Ph.D., Chief Scientific Officer of uniQure, stated that the Company is rapidly progressing its understanding and capabilities related to AAV delivery, which has the potential to significantly expand the application of its gene therapies to patients with serious metabolic liver diseases and neurocognitive disorders.

According to Sander van Deventer, uniQure believes that its expertise enables to implement novel technologies that lead to effective transduction of a wide range of target cell types in the CNS and liver.

Sander added that the Company also demonstrates the ability to express therapeutic proteins throughout the primate liver, which is mandatory for the efficacy of gene therapy for many metabolic diseases.

As per Sander, this is an important step toward uniQure's goal of delivering transformational medicine to patients suffering from genetic diseases.

About uniQure N.V.

Founded in 1998 and headquartered in Amsterdam, uniQure is delivering on the promise of gene therapy- single treatments with potentially curative results. The Company is leveraging it's modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with severe genetic diseases.

Stock Performance Snapshot

May 21, 2018 - At Monday's closing bell, uniQure's stock slightly rose 0.61%, ending the trading session at $31.50.

Volume traded for the day: 299.05 thousand shares.

Stock performance in the last three-month ? up 27.94%; previous three-month period ? up 108.75%; past twelve-month period ? up 451.66%; and year-to-date - up 60.80%

After yesterday's close, uniQure's market cap was at $1.13 billion.

The stock is part of the Healthcare sector, categorized under the Biotechnology industry. This sector was flat at the end of the session.

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