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Newsletter September 2018

News: European Commission Approves Novartis’ Kymriah CAR-T Cell Therapy
Novartis announced that the European Commission (EC) approved its chimeric antigen receptor T cell (CAR-T) cell therapy Kymriah (tisagenlecleucel) for the treatment of pediatric and young adult patients up to 25 years of age with B-cell acute lymphoblastic leukemia (ALL) and for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy.

News: Health Officials Remove Special NIH Oversite Rules Governing Gene Therapy Experiments
As gene therapies have become reality, the U.S. government is removing some special regulations that had been set up long ago over concerns of exotic safety risks. The Associated Press reported that a National Institutes of Health oversight panel will no longer be called upon to review all gene therapy applications. That panel will now take a broader advisory role, the AP said. The U.S. Food and Drug Administration (FDA) will now look at gene therapy treatments as it down all other types of medications.

Company: Arthrogen
Arthrogen is an Amsterdam based clinical stage, biopharmaceutical company, is developing local gene therapy for inflammatory diseases, using viral mediated gene transfer. The first target indication is in the field of rheumatic diseases. Currently two phase Ib trials are ongoing, one in Canada and one in the Netherlands.

Conference: Cell & Gene Therapy 2018
SMi introduces their inaugural event on Cell & Gene Therapy which takes place between the 10th and 11th October 2018 in London, with an interactive pre-conference workshop on the 9th October. This year’s event will be led by scientists and clinicians researching, developing and testing new treatments for genetically inherited and acquired diseases using gene delivery technology, stem cell manipulation and DNA repair techniques. The conference will cover all aspects of the subject, including biomedical science principles, molecular basis of disease, current and developing technologies and clinical applications.

Conference: Rare Diseases and Orphan Products Breakthrough Summit
During the 2018 NORD Rare Summit from October 15-16, 2018 in Washington, D.C., over 700 leaders from FDA, NIH, industry, patient groups, payers and research institutions will address the New Era of Patient-Focused Innovation. Together, NORD Rare Summit attendees will explore the new and innovative ways in which patients and caregivers are helping drive progress for the rare disease community.

Conference: Precision CRISPR Drug Discovery and Gene Therapy Congress
Join the Precision CRISPR Gene Editing Community in Europe. Meet and learn from thought-leaders in gene editing, like Eric Paul Bennett (University of Copenhagen), Jacob Corn (ETH Zurich), Dr Tony Perry (University of Bath), Lin Wu (Harvard) and many more at the Precision CRISPR Drug Discovery & Gene Therapy Congress in London from 22-24 October 2018. Be part of a dedicated community in Europe, striving to realise the drug discovery & gene therapy applications of optimised CRISPR system. Register now with 10% discount using code CRISPRGENT10.

Conference: World Orphan Drug Congress Europe
The 9th annual World Orphan Drug Congress (6 - 8 November 2018 in Barcelona) is the marketplace for orphan drug professionals looking at the complete value chain of orphan drug development, from clinical development and R&D to corporate development and market access. Now recognised as the largest and most established European orphan drug event, we are once again proud to deliver another outstanding speaker line-up and a content-filled agenda that represents the whole orphan drug landscape. Join us in November and make sure you are at the forefront of the orphan drug industry. Regardless of whether your interest lies in research, clinical development, patient access, global pricing and reimbursement or just to engage with patient advocacy groups more intimately, we have content, networking and potential partners for you!

If you are interested in attending make sure to register today and benefit from the 15% partner discount. Simply use the voucher code LLQF when you register online.


- 26 - 28 September 2018, XXIVth Annual Meeting of the German Society for Gene Therapy (DG-GT), Freiburg, Germany
- 10 – 11 October 2018, Cell & Gene Therapy: From manufacturing to patient access, London, UK
- 15 – 16 October 2018, Rare Diseases and Orphan Products Breakthrough Summit, Washington, DC
- 16 – 19 October 2018, XXVI Congress of the European Society of Gene and Cell Therapy (ESGCT), Lausanne, Switzerland
- 17 – 18 October 2018, Orphan Drugs and Rare Diseases Conference, London, UK
- 22 – 24 October 2018, Gene Therapy for Rare Disorders Europe, London, UK
- 22 – 24 October 2018, Precision CRISPR Drug Development and Gene Therapy Congress, London, UK
- 24 – 25 October 2018, Annual Congress on CRISPR-Cas9 Technology, Boston, MA
- 25 – 26 October 2018, 4th Annual Cell & Gene Therapy Congress, London, UK
- 6-8 November 2018, World Orphan Drug Congress 2018, Barcelona, Spain
- 8-9 November 2018, 4th Annual Genome Editing UK Congress, London, UK
- 9-10 November 2018, 2nd Annual Summit on Cell Therapy and Stem Cell Research 2018 Conference, Atlanta, GA
- 23 November 2018, British Society for Gene and Cell Therapy (BSGCT) Annual Conference Autumn 2018, London, UK
- 30 January - 1 February 2019, Gene Therapy – Ready for the Market?, Frankfurt am Main, Germany
- 6 - 7 February 2019, Annual Cell and Gene Therapy Innovation Summit, Berlin, Germany
- 7 - 8 March 2019, Spring Symposium of the Netherlands Society of Gene & Cell Therapy (NVGCT), Lunteren, The Netherlands
- 21 - 22 March 2019, 9th International Conference and Exhibition on Advanced Cell and Gene Therapy, Rome, Italy
- 3 – 5 April 2019, 4th Spring school of the European Society of Gene and Cell Therapy (ESGCT), Naples, Italy
- 8 – 10 April 2019, 6th World Congress on Human Genetics and Genetic Diseases, Abu Dhabi, UAE
- 29 April - 2 May 2019, American Society of Gene and Cell Therapy (ASGCT) 22th Annual Meeting, Washington, DC
- 25 - 26 May 2019, Gordon Research Conference: Virusses & Cells, Lucca, Italy
- 19 - 21 June 2019, Annual conference of the British Society for Gene and Cell Therapy (BSGCT), Sheffield, UK
- 20 - 23 July 2019, The 25th Annual Meeting of the Japan Society of Gene Therapy (JSGT), Tokyo, Japan
- 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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