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Newsletter May 2017

News: European Child Receives Strimvelis Gene-Therapy Treatment
Before March, only 1 patient had ever been treated via commercial gene therapy. GlaxoSmithKline (GSK) doubled that total with their announcement this week. In 2015, a European patient with familial chylomicromenia received Glybera to restore their LPL enzyme levels. On Tuesday, GSK announced that it treated its first patient in March, almost a full calendar year after their drug, Strimvelis, was approved for sale. Strimvelis is assumed to provide an outright cure for Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency (ADA-SCID), a rare disorder caused by the absence of the essential protein adenosine deaminase (ADA). ADA is required to produce lymphocytes. It leaves babies without a fully-functioning immune system and extremely vulnerable to infections.

News: uniQure Announces It Will Not Seek Marketing Authorization Renewal for Glybera in Europe
uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will not pursue the renewal of the Glybera® (alipogene tiparvovec) marketing authorization in Europe when it is scheduled to expire on October 25, 2017. "The decision to not pursue marketing authorization renewal of Glybera in Europe involved a thoughtful and careful evaluation of patient needs and the clinical use of the therapy, and is not related to any risk-benefit concern," stated Matthew Kapusta, chief executive officer of uniQure. "Glybera's usage has been extremely limited and we do not envision patient demand increasing materially in the years ahead."

News: Overcoming the limitations in gene therapy of blood cells
Transduction enhancer for Lentivirus ready for clinical applications.

Lentivirus are a heavily researched candidate for gene therapeutic applications (ex-vivo) on hematopoietic stem cells and mature T-cells. A major caveat in these approaches is that for hematopoietic cells, standard transduction efficiencies range much lower, usually between 10%-30% than common cells. Recent reports from several clinical studies confirm that the number of transduction positive cells achieved in lentivirus based therapies may correlate with therapeutic success. Finding a technique to maximize viral transduction is instrumental to overcome this challenge.

In Munich, the solution to this complex challenge is a compound named LentiBOOSTTM. The substance has been reported to push the boundaries of blood cell transductions, up to trippling efficiencies of standard techniques for blood stem and differentiated blood cells. It can be sourced in qualities that adhere to clinical safety and purity standards, making its application feasible in clinical trials. Confirmation for LentiBOOSTTM now comes from the industrial sector. SIRION Biotech and the Helmholtz Institute München recently received first milestone payments for the integration of the compound in a phase III clinical study by an industry partner. The “Munich solution” can be licensed from SIRION Biotech GmbH, Martinsried for applications in clinical R&D.


- 14 - 19 May 2017, Gordon Research Conference: Virusses & Cells, Lucca, Italy
- 17 - 19 May 2017, World Advanced Therapies and Regenerative Medicine Congress 2017, London, UK
- 24 - 26 May 2017, 10th Australasian Gene Therapy Society Meeting (AGCTS), Sydney, Australia
- 8 - 9 June 2017, 2nd Edition of Biorpocessing of Advance Cellular Therapies Congress, London, UK
- 26 - 27 June 2017, Cell Culture and Cell Therapy: Bioprocessing Conference, Philidelphia, PA
- 20 - 22 July 2017, The 23th Annual Meeting of the Japan Society of Gene Therapy (JSGT), Okayama City, Japan
- 24 - 25 August 2017, Gene Therapy Bioproduction Conference, Boston, MA
- 31 August - 1 September 2017, 2nd International Conference on Nucleic Acids, Molecular Biology & Biologics Conference, Philadelphia, PA
- 27 - 28 September 2017, Annual Summit on Cell Therapy and Molecular Medicine, Chicago, Il
- 17 – 20 October 2017, XXV Congress of the European Society of Gene and Cell Therapy (ESGCT), Berlin, Germany
- 9 - 10 November 2017, 3rd Annual Genome Editing Congress, London, UK
- 9 - 10 November 2017, 3rd Annual Cell & Gene Therapy Congress, London, UK
- 15 - 17 November 2017, 16th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases, Sonoma, California


Gene Therapy Net is the information resource for basic and clinical research in gene therapy, and the site serves as a network in the exchange of gene therapy information and breaking news items. Visitors can keep track of the latest scientific papers, conference announcements, gene therapy jobs, regulations and guidelines. Other information websites created and maintained by the owner of Gene Therapy Net are Dengue Virus Net, Influenza Virus Net, Ebola Virus Net, Chikungunya Virus Net and Zika Virus Net.
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