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Newsletter February 2018

News: High-dose AAV gene therapy appears toxic in monkeys and piglets
A gene therapy trial that recently led to dramatic benefits for babies born with a fatal neuromuscular condition has raised hopes for using a similar approach to treat other diseases. But a new animal study suggests that the high doses (2x10E14 genome copies per kilogram body weight) of gene-carrying viruses used in such treatments may not always be as safe as the human clinical trial indicated. In the new research, the disclosure of which briefly sent the stock prices of several gene therapy companies plummeting yesterday, researchers injected a handful of young monkeys and pigs with many copies of adeno-associated virus 9 (AAV9), a normally harmless virus that infects neurons and is increasingly being used to ferry therapeutic genes into cells to treat neuromuscular diseases. Within days, some of the animals developed severe liver and neuron damage.

News: Chinese scientists used Crispr gene editing on 86 human patients
China is taking the lead in the global race to perfect gene therapies. Scientists have genetically engineered the cells of at least 86 cancer and HIV patients in the country using Crispr-Cas9 technology since 2015, the Wall Street Journal reports (paywall). Although no formal scientific papers have been written about these experiments, doctors told journalists at the WSJ that some patients have improved. There have also been at least 15 deaths, although only about half of them were reportedly related to the gene therapy itself. These therapies, which involved taking the immune cells from hospital patients, editing the cells, and transfusing them back into the body, are the first to use Crispr-Cas9 in living humans.

Conference: Precision CRISPR Congress 2018
The 4th Precision CRISPR Congress is taking place in Boston on February 26-28, 2018 - the worlds leading industry focused, commercially driven meeting focused on optimizing the next generation applications of CRISPR. United by the common goal of increasing efficiency, optimizing delivery and reducing known off-target effects, the Precision CRISPR Congress will host two distinct tracks that delve deeper into the biomedical research and therapeutic applications of CRISPR, as well as highlighting the translational steps needed to effectively advance CRISPR into the clinic.
Promotional Code: ‘genetherapy10’ for 10% off all tickets.

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Conference: Patients as Partners US
The 5th Annual Patients as Partners program is the only conference (March 15-16, 2018 in Philadelphia) in the US that demonstrates how to involve patients throughout the entire medicines development life-cycle. It 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.
Promotional Code: ‘GTN15’ for 15% off all tickets.

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Conference: CAR-T Congress USA
CAR-T was the star of 2017, register by 12.01.2018 & save $400 to hear more about its future Following a successful year of FDA approvals in both USA and Europe, along with Gilead’s recent acquisition of Cell Design Labs, as well as Celgene and bluebirdbio’s latest launch into their next phase of their BMCA-targeting study…2018 should be a pivotal year for CAR-T! Join 25+ industry leaders on March 20-22, 2018 to learn more about the latest innovations in manufacturing, use of alternative CARs constructs, as well as how to tackle solid tumors at the CAR-T Congress USA.
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Conference: Gene Therapy for Rare Disorders 2018
Gene Therapy for Rare Disorders 2018 (April 30 – May 2, 2018, Boston, MA) will focus exclusively on overcoming the late-stage commercial challenges drug developers face when delivering gene therapies to market. Incorporating insights from Spark, Bluebird Bio, Pfizer and GSK, this unique conference will delve into the key regulatory, reimbursement, clinical and manufacturing hurdles that need to be overcome to realize the commercial potential of gene therapies. Join your colleagues to accelerate the progress of the next generation of gene therapies that show improved efficacy, enhanced safety and commercial viability.
Gene Therapy Net readers receive an exclusive 20% discount when quoting 9536GTN at registration.

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- 23 - 24 January 2018, Patients as Partners Europe Conference, London, UK
- 26 - 28 February 2018, Precision CRISPR Congress 2018, Boston, MA
- 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 - 16 March 2018, 7th International Conference and Exhibition on Cell and Gene Therapy, London, UK
- 20 - 22 March 2018, CAR-T Congress USA, Boston, MA
- 21 - 23 March 2018, ESGCT Spring School 2018, Oxford, UK
- 9 - 12 April 2018, 11th International Oncolytic Virus Conference Oxford, UK
- 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
- 30 April - 2 May 2018, Gene Therapy for Rare Disorders Summit, Boston, MA
- 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
- 18 - 19 June 2018, International conference on Stem Cells and Regenerative Medicine - Genome editing for targeting genetic diseases, Osaka, Japan
- 23 - 24 July 2018, Annual Congress on Advanced Tissue Science and Regenerative Medicine, Rome, Italy
- 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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