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Newsletter April 2019

News: First gene therapy to treat rare blood disease nears European approval
The first gene therapy to treat a rare blood disorder is one step closer to approval Friday following a recommendation by European officials. Lentiglobin, the gene therapy for beta-thalassemia developed by Cambridge, Mass.-based Bluebird Bio, was recommended for approval by the Committee for Medicinal Products for Human Use (CHMP), the drug-reviewing arm of the European Medicines Agency. A final approval decision is expected within the next three months.

News: Should gene therapy patients be asked to pay?
The price in value-based payment deals should by definition be justified, so why is patient cost-sharing part of the calculation? Spark covers this cost, but there is no guarantee that all gene therapy companies will. As Novartis’s gene therapy Zolgensma nears a US FDA decision, the sector has only a vague idea of how much the Swiss group will charge for the spinal muscular atrophy (SMA) treatment, other than it will be a number followed by six zeroes. Just as importantly, the families with children suffering from this fatal condition do not know what, if anything, they will be asked to pay. However, there are some clues from the way in which the first US-approved gene therapy, Spark Therapeutics’ Luxturna, was financed.

Conference: NDD Europe 2019
Overcome Translational Challenges Preventing the Development of Clinically Effective Neurodegenerative Therapeutics. Multiple clinical failures in the neurodegenerative field have sparked realisation that there is a critical need for discussion and debate between neurodegenerative drug developers to drive clinical success.
NDD Europe, 21st – 23rd May, 2019 in Paris, will overcome the translational challenges facing neurodegenerative drug developers by exploring the future biomarkers of neurodegeneration, improving preclinical modelling to increase clinical predictability and encouraging precompetitive strategies and collaboration amongst drug developers.
With 70+ thought-leaders converging at NDD Europe, this will be an unrivalled opportunity for you to gain the latest scientific insights, have your peers review your research and grow your collaborator network


Conference: 4th PDA Europe Annual Meeting
Heading to its 4th edition, PDA Europe Annual Meeting, 25-26 June, 2019 in Amsterdam, continues the aim to identify ways of improving patients‘ health and welfare by sharing best-practice examples, experiences, novel approaches and professional insights from stakeholders along the entire path, starting with R&D, to manufacturing, storage, distribution, and finally clinical application, payers‘ perspectives and patient needs. We grant 20% discount to maximum 10 people, here is the code: 2019 PDA-EU_ANNUAL. The code should be placed on a registration form and sent via e-mail

- 23 – 25 April 2019, The Cell & Gene Meeting on the Mediterranean, Barcelona, Spain
- 29 April - 2 May 2019, American Society of Gene and Cell Therapy (ASGCT) 22th Annual Meeting, Washington, DC
- 7 - 9 May 2019, Allogeneic Cell Therapies Summit 2019, Boston, MA
- 14 - 15 May 2019, Cell Series US Congress: Cell Culture & Bioprocessing and Cell & Gene Therapy, Boston, MA
- 14 - 16 May 2019, Cell and Gene Therapy, Shanghai, China
- 15 - 17 May 2019, World Advanced Therapies & Regenerative Medicine Congress 2019, London, UK
- 21 - 23 May 2019, 5th Annual Precision CRISPR Congress Boston, Boston, MA
- 21 - 23 May 2019, NDD Europe - Neurodegenerative Drug Development - Focus on gene therapy, Paris, France
- 25 - 26 May 2019, Gordon Research Conference: Virusses & Cells, Lucca, Italy
- 4 - 5 June 2019, Advanced Therapy Medicinal Products conference, Vilnius, Lithuania
- 18 - 20 June 2019, CAR-TCR Summit Asia 2019, Shanghai, China
- 18 - 20 June 2019, Next generation CAR & T Cell Therapies, San Francisco, CA
- 19 - 21 June 2019, Annual conference of the British Society for Gene and Cell Therapy (BSGCT), Sheffield, UK
- 25 - 26 June 2019, 4th PDA Europe Annual Meeting - gene therapy regulatory issues, Amsterdam, The Netherlands
- 20 - 23 July 2019, The 25th Annual Meeting of the Japan Society of Gene Therapy (JSGT), Tokyo, Japan
- 9 - 12 September 2019, Cell & Gene Therapy Manufacturing and Commercialization, Boston, MA
- 10 - 13 September 2019, CAR-TCR Summit Boston 2019, Boston, MA
- 9 October 2019, The Cell & Gene Therapy Strategy Meeting Europe, Zurich, Switzerland
- 21 – 25 October 2019, XXVII Congress of the European Society of Gene and Cell Therapy (ESGCT), Barcelona, Spain


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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