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Newsletter December 2013

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News: UK's first gene therapy trial for patients with chronic heart failure begins at GJNH
The UK’s first gene therapy trial for advanced heart failure, CUPID 2, has officially begun at the Golden Jubilee National Hospital (GJNH); with the first candidate recently being administered with a dose of the MYDICAR treatment. The GJNH, home of the Scottish National Advanced Heart Failure Service (SNAHFS), announced in April 2013 that it would be participating in the international trial, helping gather a total of 200 patients from 50 institutions worldwide, to determine the effectiveness of the gene therapy treatment, MYDICAR, in advanced heart failure patients.

Explainer: what is gene therapy?
Every now and again you might read about gene therapy and efforts to correct serious genetic diseases. But I’m betting that very few readers have had gene therapy, nor have they ever met anyone who has, nor will they ever meet that many. The reasons are simple – while these procedures are possible in theory, in practice, it has proved remarkably difficult to insert new genetic materials into human patients and ensure that the new genes are sustainably expressed. The difficulties encountered highlight the fact that we do not yet have enough fundamental knowledge about gene transfer and control. And what’s more, gene therapy is hugely expensive.

Q&A: Gene Therapy the Fix for Faulty DNA
Inside (almost) all of our cells are 23 pairs of chromosomes, long strands of DNA that are packaged tightly together. Each chromosome houses hundreds of genes, each of which is the blueprint for at least one protein. Little messengers called mRNA make copies of the gene blueprints, edits them and carry them to “builders” called ribosomes. The ribosomes read the copy and translate it into long chains of amino acids that are then folded into unique shapes. These globs of amino acids are proteins, that travel throughout our entire body and are in some way vital for everything that happens inside of us. For example, helicase and chromatin are proteins essential to cell division, what allows us to grow and heal, and immunoglobulin, also called antibodies, protect us from infections.
What happens when something goes wrong with our DNA and thus our proteins?


Conference: Cell & Gene Therapy 2014
The Phacilitate Cell & Gene Therapy Forum is celebrating its 10th anniversary and goes from strength-to-strength, mirroring the performance of the sector itself. More big pharma speakers and more regulators than ever before: including senior representatives from Pfizer, J&J, GSK, Novartis, Sanofi, Roche, Baxter, Biogen Idec, EMA, FDA, PMDA and Cigna.

The ever popular annual sector progress report returns with 2013 top 10 revealed - a countdown of the year’s most significant events in C>, and the 2012 top 10 revisited – how have they advanced in 2013? The 2014 agenda is packed with 80+ speakers, 5 plenary sessions offering commercialization and business insights plus 9 focus sessions on characterisation and potency assays, pre-clinical and clinical development, regulatory and manufacturing challenges, plus case studies in specific therapeutic areas - namely oncology, blood disorders, cardiovascular diseases and rare/orphan conditions.

BRAND NEW FOR 2014: co-located with Stem Cells as Discovery & Research Tools 2-day all-plenary conference specifically designed to provide the strategic insights and networking opportunities required by senior decision makers from those companies that provide stem cell tools for drug discovery, as well as those who utilize them. Speakers representing NIH CRM, CIRM, IMI StemBancc, Pfizer, Janssen, Roche and Celgene head up the discussions which span business models and collaborations, regulatory updates and cutting edge applications.

Dates: 27-29 January 2014
Venue: Washington DC, USA


Conference: Gene-based Therapeutics for Neurological Disorders: From Basic Science to Clinical Applications
There are still some oral slots available for our forthcoming ‘’Gene-based Therapeutics for Neurological Disorders: From Basic Science to Clinical Applications” conference, 8-11 February 2014 at the Fiesta Americana Condesa Resort, Cancun, Mexico.

This is a great opportunity to assemble a rich programme of international speakers to address the latest advances in gene therapy, from basic research of vector design, modelling and pre-clinical testing to clinical applications in CNS disorders such as Parkinson’s, Alzheimer’s, Huntington’s, motor neuron diseases and eye diseases.

The conference will bring together basic and clinical scientists to discuss recent discoveries in genetics and mechanisms of disease (e.g. the role of RNA metabolism and processing in neurological diseases, including amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), spinal muscular atrophy (SMA), and repeat expansion disorders) and how these discoveries could help develop novel gene based therapeutics for neurodegenerative diseases. Further topics such as challenges in vector manufacturing and views of regulatory agencies on gene therapy clinical applications will be discussed.

Plenary Speakers include: Professor Richard Jude Samulski (University of North Carolina) Professor Jeffrey H. Kordower (Rush University, Chicago) and Professor Ronald G Crystal (Weill Medical College of Cornell University).

Invited Speakers include: Dr Krystof Bankiewicz (University of California), Dr Howard Federoff (Georgetown University, Dr Maria Kinali (Imperial College, London), Dr Brian Kaspar (Nationwide Children's Hospital, Columbus) and Dr Fraser Wright (Childrens Hospital of Philadelphia).

If you are interested in presenting orally or via a poster, please register for the conference to ensure ample time for abstract consideration. If you are looking to only attend, please register with us as soon as possible to ensure you secure a place. Registration closes on 13 December 2013: Register now

Dates: February 8-11, 2014
Venue: Cancun, Mexico


- 27 - 29 January 2014, Phacilitate 10th Annual Cell & Gene Therapy Forum 2014, Washington DC, MD
- 8 -11 February 2014, Gene-based Therapeutics for Neurological Disorders, Cancun, Mexico
- 13 - 14 March 2014, Spring Symposium of the Netherlands Society of Gene & Cell Therapy (NVGCT), Lunteren, The Netherlands
-20 -22 March 2014, 20th Annual Meeting of the German Society for Gene Therapy (DG-GT e.V.), Ulm, Germany
- 24 - 26 March 2014, French Society of Cell and Gene Therapy (SFTCG) Annual Congress 2014, Toulouse, France
- 28 March 2014, Annual conference of the British Society for Gene Therapy (BSGT), London, UK
- 21 – 24 May 2014, American Society of Gene and Cell Therapy (ASGCT) 17th Annual Meeting, Washington, DC
- 6 – 8 August 2014, The 20th Annual Meeting of the Japan Society of Gene Therapy (JSGT), Tokio, Japan
- 11 – 13 August 2014, The 2nd International Conference on Genetic Engineering & Genetically Modified Organisms, San Antonio, TX
- 25 – 27 September 2014, 14th Annual Meeting of the International Society for Cell & Gene Therapy of Cancer, Amsterdam, The Netherlands
- 23 – 26 October 2014, XXII Congress of the European Society of Gene and Cell Therapy (ESGCT), The Hague, The Netherlands
- 27 – 29 October 2014, 3rd International Conference and Exhibition on Cell & Gene Therapy, Las Vegas, NV
- November 2014, Formulation and Drug Delivery Congress, London, UK
- November 2014, Cell Therapy Congress, London, UK


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