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Newsletter December 2019

News: Hopkins team invents non-viral system for getting gene therapy into cells
One of the most popular methods for inserting therapeutic genes into cells to treat disease is to transport them using a virus that has been stripped of its infectious properties. But those noninfectious viruses can still sometimes touch off dangerous immune responses. A team from Johns Hopkins Medicine is proposing an alternative method for transporting large therapies into cells—including genes and even the gene-editing system CRISPR. It’s a nano-container made of a polymer that biodegrades once it’s inside the cell, unleashing the therapy. The researchers described the invention in the journal Science Advances.

News: Encouraging early results from first human CRISPR gene therapy trials
Promising preliminary data from one of the first human trials testing the safety and efficacy of a CRISPR gene therapy has just been revealed. Although it is too early to evaluate long-term effects, the initial reports are impressively successful for two patients with severe genetic blood diseases. Until February of this year, when pharmaceutical companies CRISPR Therapeutics and Vertex began a large global trial into a treatment called CTX001, no human outside of China had been officially treated with a CRISPR-based gene editing therapy. CTX001 was developed to treat two types of inherited blood disease, beta-thalassemia and sickle cell disease. Both conditions are caused by a mutation in a single gene and the treatment involves engineering a patient's stem cells with a single genetic change designed to raise levels of fetal hemoglobin in red blood cells.

Conference: Cell & Gene Therapies pipeline at PepTalk 2020
The inaugural Cell & Gene Therapies pipeline at PepTalk 2020 (January 20-24 in San Diego, CA) features three back-to-back conferences exploring the challenges, advances, and opportunities in gene and cell therapies. The conferences on Vector Design and Development for Gene and Cell Therapies, Gene Therapy Analytics and Manufacturing, and Cell Therapy Analytics and Manufacturing will discuss critical and hot topics like engineering and development of safer and more efficacious vectors, CMC and characterization, manufacturing, scale-up, next-generation production technologies, automation, supply chain, commercialization, and many more practical issues for the success of cell and gene therapies.
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Conference: 11th Annual RNA Therapeutics Conference
SMi Group are pleased to present the 11th Annual RNA Therapeutics Conference and Focus Day in London on February 18th - 20th 2020.
The field of RNA therapeutics is rapidly expanding, and the potential for using RNA drugs for personalised medicines and immunotherapy, as well as to address genetic, infectious and chronic diseases will ensure the continued development of RNA therapeutics for years to come.
The event will focus on the latest research and developments on delivery system technologies that aim to tackle more difficult targets within the human body. This overarching topic warrants high focus and therefore, for the first time in the series, introduce a focus day on Oligonucletide Delivery Systems. The main conference will focus on current developments in the area of mRNA therapeutics in immunotherapy and vaccination.

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Conference: CAR-TCR Europe Summit
With CAR-T therapies on the cusp of achieving global approval, there are still many bottlenecks that are preventing this from becoming the ‘sell-out’ therapy that the field had hoped for. The CAR-TCR Europe Summit (February 24-27, 2020 in London) will unravel the technical challenges across translation, scale and delivery to provide your team with the platform to learn, collaborate and gain actionable insights to advance your therapy for clinical and commercial success.
This focused agenda will delve into the technical bottlenecks encountered in every stage of the CAR-TCR drug development cycle, providing a comprehensive analysis across 3 tracks including: Translational, Manufacturing & Clinical.

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Conference: Neurodegenerative Drug Development Summit 2020
Returning to Boston in February 2020 is the 8th Neurodegenerative Drug Development Summit in Boston this February 25-27. Despite the lack of clinical trial success, new investments are being made in forward-thinking biotechs, digital and blood-based biomarker technologies are advancing, new targets are emerging, and gene therapy is taking the limelight.
The Neurodegenerative Drug Development Summit will unite industry stakeholders to learn how to overcome their translational challenges, share their progress and network for future collaborations to accelerate the development of successful neurodegenerative therapeutics.
Don’t miss your opportunity to join over 150 CNS expert from Biogen, AbbVie, Sanofi and more, and immerse yourself in the neurodegenerative community to help refresh the neurodegenerative approach and challenge traditional thinking to advance clinical progress.
In partnership with the meeting, grab an additional 10% off with the code “GTN10” when you register online.

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Conference: 6th annual Immuno-Oncology 360° Summit
IO360 ̊ convenes stakeholders spanning the science and business communities to report on the latest data impacting immuno-oncology to fight a wider range of cancers.
The 6th annual Immuno-Oncology 360° Summit (February 26-28, 2020 in New York) is the premier meeting across all stakeholders in IO, addressing the rapid advancements of clinical, scientific and business developments of IO in one setting to help develop therapeutics for a wider range of cancers at an accelerated rate.
The 3 day summit features more than 80 time sensitive IO topics, over 500 senior level thought leaders and includes over 10 hours of networking. New for 2020: Cell & Gene Therapy Day - A full day on February 27th dedicated to Cell & Gene Therapy for Solid Tumors. Chaired by Dr Aiman Shalabi, GSK. Keynotes, Cornerstone and Notable Speakers include Dr Carl June, UPenn, Dr Crystal Mackall, Stanford University, Dr Arie Belldegrun, Allogene, Dr Peter Marks, FDA, Dr Stephanie Goff, NCI, amongst many more.
Use the code GTN15 for a 15% discount.

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- 20 - 24 January 2020, PepTalk event on Cell and Gene Therapies, San Diego, CA
- 29 - 30 January 2020, CAR-T Congress EU, London, UK
- 29 - 30 January 2020, Vein-to-Vein Congress EU - Manufacturing, scale-up, regulatory and logistical bottlenecks to commercialise cell therapies, London, UK
- 5 - 6 February 2020, Annual Cell and Gene Therapy Innovation Summit, Berlin, Germany
- 19 - 20 February 2020, RNA Therapeutics 2020, London, UK
- 22 - 24 February 2020, CAR-TCR Summit Europe, London, UK
- 25 - 27 February 2020, Neurodegenerative Drug Development Summit 2020 - Gene Therapy, Boston, MA
- 26 - 28 February 2020, Immuno-Oncology 360° - Cell & Gene Therapy Day, New York, NY
- 2 - 3 March 2020, Tri-Conference 2020 - Renaissance of Gene Therapy and Genome Editing, San Francisco, CA
- 3 - 5 March 2020, Gene Therapy for Blood Disorders, Boston, MA
- 23 - 25 March 2020, International Society for BioProcess Technology 10th Spring Meeting - Viral vectors and vaccines, Norfolk, Virginia
- 24 - 26 March 2020, Innate Killer Summit 2020, San Diego, CA
- 24 - 26 March 2020, Bioprocessing Summit Europe, Barcelona, Spain
- 30 March - 2 April 2020, 4th Annual Gene Therapy for Rare Disorders, Boston, MA
- 31 March - 2 April 2020, 15th Annual World Advanced Therapies Congress, London, UK
- 7 - 8 April 2020, 4th Annual Genome Editing USA Congress, Boston, MA
- 15 - 16 April 2020, Global Cell and Gene Therapy Innovation Leaders Shanghai Forum - Asia Pharma R&D Leaders Summit 2020, Shanghai, China
- 22 - 23 April 2020, Formulation and Drug Delivery conference - Cell & Gene Therapy Biomanufacturing: Insights, Key Strategies & Novel Approaches, London, UK
- 29 - 30 April 2020, CAR-T Congress USA, Boston, MA
- 12 - 15 May 2020, American Society of Gene and Cell Therapy (ASGCT) 23th Annual Meeting, Boston, MA
- 15 - 19 June 2020, 9th International Symposium on Monolith - DNA/RNA, vaccines, gene therapy vectors, Portoroz, Slovenia
- 20 - 23 July 2020, 2nd Drug Delivery Conference Bridging the Gap between Basic Science and Unmet Clinical Needs, Lisbon, Portugal
- 24 - 28 August 2020, 12th annual Bioprocessing Summit, Boston, MA
- 14 - 17 September 2020, CAR-TCR Summit USA, Boston, MA
- 20 – 23 October 2020, XXVIII Congress of the European Society of Gene and Cell Therapy (ESGCT), Edinburgh UK


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