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Newsletter December 2017

News: Alarmed by Recent Events, Scientists Speak Out Against DIY Gene Therapy
It seems hard to fathom that scientific progress has advanced to a point where it’s suddenly possible for people to undertake something as complicated as gene therapy outside the safety and confines of a well-funded lab, but it has. In just the past few months we’ve seen not one, but two examples of people pursuing unregulated gene therapy. That’s right, people are genetically modifying their bodies in DIY labs and friend’s apartments. Now, the largest organization of scientists who work in gene and cell therapy have come out against DIY gene therapies. A statement from the American Society of Gene and Cell Therapy comes on the heels of a stern warning against DIY gene therapy from the US Food and Drug Administration. For years, when it came to biohacking, regulators and establishment science have mainly looked the other way. That seems to be changing.

News: FDA warns against ‘do it yourself’ gene therapy kits
The U.S. Food and Drug Administration has issued a stern warning against the use of “do it yourself” gene therapy kits, pushing back against a nascent “biohacker” movement that seeks to make experimental medicines and technologies available to the masses, often by circumventing regulators. A statement posted on the FDA’s website on Tuesday cautions that it is illegal to sell gene therapy products and kits intended for self-administration. Gene therapy involves inserting new, healthy genes into cells to cure diseases that are caused by faulty genes. The FDA has classified gene editing — cutting out and replacing parts of faulty genes — as a form of gene therapy. A host of local biotechs, including Cambridge-based Editas Medicine (Nasdaq: EDIT), are seeking to develop such treatments.

News: First gene-editing attempted in human body to cure disease
Doctors have edited a patient's genes in a world-first attempt to alter DNA from within the human body. Medics in California administered the experimental intravenous treatment, which provides the body with instructions to change DNA, on Brian Madeux, who suffers from Hunter's syndrome. People with Hunter's syndrome are unable to break down long sugary molecules called mucopolysaccharides. This leads to the molecules building up and causing brain and organ damage, and in severe cases it can be fatal. Sufferers require regular enzyme replacement therapy to break down the molecules, but Mr Madeux said he opted to take part in a trial as he is "in pain every second of the day". The 44-year-old from Arizona underwent the treatment at UCSF Benioff Children's Hospital in Oakland, California on Monday.

News: Gene therapy helps a boy get new skin
A medical team at the Ruhr-Universität Bochum’s burn unit and the Center for Regenerative Medicine at the University of Modena (Italy) were the first ever to successfully treat a child suffering from extensive skin damage using transplants derived from genetically modified stem cells. The boy is a so-called butterfly child: he suffers from epidermolysis bullosa, a genetic skin disease that had destroyed approximately 80 percent of his epidermis. After all established therapies had failed, the medical team from Bochum decided to try an experimental approach: they transplanted skin derived from genetically modified stem cells onto the wound surfaces. Thanks to the successful therapy, the boy is now – two years after the treatment – able to participate in his family’s life and social life. The scientists published their report in “Nature”.

Conference: CAR-TCR Summit Europe
To date, the clinical results in solid tumour trials have been lack lustre. There are several aspects of development that need to be improved in the next generation of CAR-T and TCR approaches to ensure success.

What is the biggest barrier to success?
“I think we, like everybody else, are struggling with how to engage T cells in the solid tumour micro environment. And so I think that remains a tremendous opportunity for the field but one which is daunting scientifically at this point.” – Philip Gregory, CSO, bluebird bio (Speaker at CAR-TCR Summit in Boston, 2017)

CAR-TCR Summit Europe, the leading end-to-end summit focused on the global delivery of safe, effective and commercially successful CAR and TCR T cell immunotherapies, will address how we can improve the underwhelming performance of CAR-TCR therapies in solid tumour indications, as well as discuss European regulations and global distribution challenges. Taking place in London, UK (20-22 February) it brings you access to the leaders in this field and the hub of companies creating novel innovations in Europe, setting the stage for visionary leaders to transform the commercial and clinical delivery of cancer therapeutics.

You will get the chance to gain exclusive insight on current clinical programs and case studies on the wave of next generation CAR-TCR candidates from those pioneering in this field. Focusing on navigating the regulatory minefield, ensuring successful global distribution and successful delivery of CAR-T and TCR therapies in solid tumours, the CAR-TCR Summit Europe is your opportunity to network, interact and learn at the only end-to-end meeting that fosters the transformation of leading science to commercial therapies.


Conference: 2nd Annual Patients as Partners Europe
Patients as Partners EU is co-produced with patients, industry, academia, government and nonprofit organizations to establish a well-rounded program that addresses the needs of all stakeholders seeking to implement and advance patient involvement across the entire clinical development continuum. Each session puts ideas into action and demonstrates the what, where, when and how behind the topic. The sessions demonstrate how these insights were used to co-create solutions with patients and measure the impact of those initiatives.

The Conference Forum is pleased to announce that the 2nd Annual Patients as Partners Europe program is officially Patients Included accredited.

Register now with code 'GTN15' and get a 15% discount on registration.


- 23 - 24 January 2018, Patients as Partners Europe Conference, London, UK
- 23 - 25 January 2018, Cell & Gene Therapy World event 2018, Miami, Fl
- 30 - 31 January 2018, Combined CAR-T Congress Europe, Berlin, Germany
- 7 - 8 February 2018, Annual Cell and Gene Therapy Innovation Summit, Berlin, Germany
- 8 - 9 February 2018, 3rd Annual Genome Editing & Engineering Conference, San Diego, CA
- 28 February - 1 March 2018, Cell Therapy Manufacturing Asia 2018, Kyoto, Japan
- 5 - 7 March 2018, International Society for BioProcess Technology 8th Spring Meeting - Viral vectors and vaccines, Norfolk, Virginia
- 14 – 16 March 2018, 9th Biennial Congress of the Spanish Society of Gene (SETGyC) and Cell Therapy, Palma de Mallorca, Spain
- 15 March 2018, British Society for Gene and Cell Therapy (BSGCT) Public Engagement 2018, Oxford, UK
- 15 - 16 March 2018, Spring Symposium of the Netherlands Society of Gene & Cell Therapy (NVGCT), Lunteren, The Netherlands
- 15 - 17 March 2018, 7th International Conference and Exhibition on Cell and Gene Therapy, London, UK
- 20 - 22 March 2018, CAR-T Congress USA, Boston, MA
- 25-26 April 2018, Global Experts Meeting on Cell and Gene Therapy, Dubai, UAE
- 27 April 2018, Annual conference of the British Society for Gene and Cell Therapy (BSGCT), Cardiff, Wales, UK
- 2 - 5 May 2018, International Society for Cellular Therapy (ISCT) Annual Meeting, Montreal, Canada
- 10 - 11 May 2018, 2nd Annual Genome Editing USA Congress, Boston, MA
- 16 - 19 May 2018, American Society of Gene and Cell Therapy (ASGCT) 21th Annual Meeting, Chicago, IL
- 15 - 20 June 2018, Monolith Summer School and Symposium (MSS) - Bioprocessing of macromolecules and viruses, Portorož, Slovenia
- 26 - 28 July 2018, The 24th Annual Meeting of the Japan Society of Gene Therapy (JSGT), Tokyo, Japan
- 13 - 16 August 2018, Gene Therapy Bioproduction Conference, Boston, MA
- 16 – 19 October 2018, XXVI Congress of the European Society of Gene and Cell Therapy (ESGCT), Lausanne, Swizterland
- 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
- November 2018, 4th Annual Genome Editing UK Congress, London, UK
- 29 April - 2 May 2019, American Society of Gene and Cell Therapy (ASGCT) 22th Annual Meeting, Washington, DC
- 28 – 31 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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