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Newsletter December 2018

News: Chinese Researchers Claim to Have Genetically Engineered the First HIV-Immune Babies
Researchers in China (Clinical project 'Safety and validity evaluation of HIV immune gene CCR5 gene editing in human embryos') used genetic engineering tools (CRISPR) to create twins theoretically immune to HIV, smallpox, and cholera, MIT Technology Review reported. The medical breakthrough is controversial, as many worry about eugenics and designer babies for the wealthy. The twins, named Lulu and Nana, according to lead scientist He Jiankui of Shenzhen in a YouTube video, were the result of in vitro fertilization (IVF). A few weeks old, they appear to be healthy. When they were a single cell, genetic surgery using a popular tool, CRISPR, “removed the doorway through which HIV enters to infect people.”
Watch the presentation of He Jiankui and discussion during Second International Summit on Human Genome Editing in Hongkong, Wednesday November 28th, 2018.


News: Spark Therapeutics' Luxturna Gets EU Approval for Vision Loss
Spark Therapeutics, Inc. announced that the European Commission has approved the marketing authorization application ("MAA") for its gene therapy, Luxturna (voretigene neparvovec). The MAA sought approval for the therapy in adult as well as pediatric patients as a one-time treatment for vision loss due to inherited retinal dystrophy caused by a genetic mutation in both copies of the RPE65-gene and who have enough viable retinal cells. The RPE65 mutations, a progressive disease, can lead to total blindness.

Conference: CAR-T Congress EU
CAR-T cell therapy has shown unparalleled success in haematological malignancies, paving the way for the field of immuno-oncology. In 2017 two therapies achieved FDA approval in the USA, and following recent approval by the EMA, it is a landmark time for the industry.

Despite the huge successes there are still a number of hurdles to overcome before CAR-T can be effective in mainstream oncology beyond haematological malignancies. The European CAR-T Congress’ mission is to address the opportunities and challenges that presently face the CAR-T field, to maximise the potential of this ground-breaking therapy.

The CAR-T Congress EU takes place from 30 - 31 January, 2019 in London, UK

Register now...

Conference: 10th Annual RNA Therapeutics
The field of RNA therapeutics has seen rapid development as RNA drugs are being utilised for personalised medicines and immunotherapy, and to address genetic, infectious and chronic diseases. This year's 10th annual conference on RNA Therapeutics is set to address the latest advancements and challenges in the industry as a host of scientific pioneers and senior experts gather in London (20-21 February, 2019) to share their extensive knowledge and expertise.

Download the latest brochure to see the full agenda and speaker line-up!


Conference: CAR-TCR Summit Europe
The industry-focused CAR-TCR Summit Europe takes place from 25-27 February, 2019, in London and is dedicated to developing, translating and delivering next generation CAR and TCR therapies for patients in Europe. Join over 250 of Europe’s leading drug developers to learn about never-before-seen clinical updates and novel technology to improve solid tumour targeting, advance allogeneic therapy development and automate manufacturing processes in order to create a targeted, efficacious and commercially viable cancer therapy.

For more information, visit


Conference: Therapeutic Genome Editing Congress
As the race to commercial genome editing therapies accelerates, the pressure is on to find innovative solutions to critical challenges in safety and delivery. We are pleased to announce the launch of the Therapeutic Genome Editing Congress in Boston on 20th-21st March, 2019.

Rapid development has seen the first clinical applications of CRISPR, TALENS and ZFNs appearing where the risk-benefit ratio is ethical. With new technologies to eliminate off-targets and innovations in delivery, its time to build a road map to market approval!

You are invited to join us as we bring together bring together peers from biotech, technology developers, pharma, and academia along with experts from government bodies, medical institutions and commercial consultants, to discuss how to progress clinical development.


- 30 - 31 January 2019, CAR-T Congress EU, London, UK
- 30 - 31 January 2019, Gene Therapy – Ready for the Market?, Frankfurt am Main, Germany
- 6 - 7 February 2019, Annual Cell and Gene Therapy Innovation Summit, Berlin, Germany
- 20 - 21 February 2019, 10th Annual RNA Therapeutics Conference, London, UK
- 25 - 27 February 2019, CAR-TCR Summit Europe 2019, London, UK
- 4 - 6 March 2019, International Society for BioProcess Technology 9th Spring Meeting - Viral vectors and vaccines, Norfolk, Virginia
- 7 - 8 March 2019, Spring Symposium of the Netherlands Society of Gene & Cell Therapy (NVGCT), Lunteren, The Netherlands
- 19 - 21 March 2019, 2nd Annual Bioprocessing Summit Europe - Cell and Gene Therapy, Lisbon, Portugal
- 20 - 21 March 2019, The Therapeutic Genome Editing Congress, Boston, MA
- 21 - 22 March 2019, 9th International Conference and Exhibition on Advanced Cell and Gene Therapy, Rome, Italy
- 26 - 28 March 2019, 3rd Annual Gene Therapy for Rare Disorders, Boston, MA
- 2 – 4 April 2019, CAR-T Congress USA, Boston, MA
- 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
- 14 - 15 May 2019, Cell Series US Congress: Cell Culture & Bioprocessing and Cell & Gene Therapy, Boston, MA
- 15 - 17 May 2019, World Advanced Therapies & Regenerative Medicine Congress 2019, London, UK
- 25 - 26 May 2019, Gordon Research Conference: Virusses & Cells, Lucca, Italy
- June 2019 CAR-TCR Summit Asia 2019, Singapore
- 4 - 5 June 2019, Advanced Therapy Medicinal Products conference, Vilnius, Lithuania
- 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
- 9 - 10 September 2019, The Gene Therapy 2019 - Novel Approaches in Human Genome and Genetic Disorders, London, UK
- 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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