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Newsletter March 2017

News: Doctors reverse teen's sickle cell disease with innovative gene therapy
A 16-year-old boy in France has become the first person ever to be successfully treated with a new first-of-its-kind gene therapy for sickle cell disease. Doctors removed the teen's bone marrow and genetically altered it in the laboratory to correct the defect in his DNA that causes sickle cell disease. The study stated 15 months after the very first treatment that the boy who once had significant internal damage - so much so that it led to a removed spleen and a hip replacement - now has no sign of the disease.

Conference: Oxford Global’s Genome Editing and Advances in Transgenic Technology USA Congress

Taking place on 10-11 May 2017 in Boston USA this co-located congress will attract 250+ delegates representing the leading research & academic institutions and clinical research institutions in the USA, as well as leading pharmaceutical and biotech companies from around the globe. Across the two days streams will focus on: • Genome Editing Techniques
• Therapeutic Applications of Genome Editing
• Drug Discovery & Development Applications
• Transgenic Genome Editing Techniques
• Advances in Animal Biotechnology: Discovery & Development
• Generation of Transgenic Models – Models of Disease and Applications
• Genetics, Epigenetics, Stem Cell Manipulation
• Technology Workshop: Novel Transgenic Technologies

Contact Danielle Dalby today for further information on This email address is being protected from spambots. You need JavaScript enabled to view it. or download the full congress programmes.


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.


- 16 - 17 March 2017, Spring Symposium of the Netherlands Society of Gene & Cell Therapy (NVGCT), Lunteren, The Netherlands
- 17 March 2017, British Society for Gene and Cell Therapy (BSGCT) Public Engagement 2017, Oxford, UK
- 27 - 28 March 2017, 6th International Conference and Exhibition on Cell and Gene Therapy, Madrid, Spain
- 29 - 30 March 2017, 19th International Conference on Cell and Gene Therapy - ICCGT 2017, Singapore
- 19 - 21 April 2017, Annual conference of the British Society for Gene and Cell Therapy (BSGCT), Cardiff, Wales, UK
- 24 - 26 April 2017, Gene Therapy for Rare Disorders 2017, Boston, MA
- 3 - 6 May 2017,International Society for Cellular Therapy (ISCT) 23th Annual Meeting, London, UK
- 10 - 11 May 2017, Genome Editing & Advances in Transgenic Technology USA Congress, Boston, MA
- 10 - 13 May 2017, American Society of Gene and Cell Therapy (ASGCT) 20th Annual Meeting, Washington, DC
- 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


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