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Newsletter January 2020

News: Dog Study Revives Concerns About Virus Used for Gene Therapy
Adeno-associated viruses are popular vectors for delivering gene therapies to patients’ cells because, researchers believed, the DNA these viruses carry rarely inserts into the host genome. But according to results presented last month (December 9) at the American Society of Hematology (ASH) meeting in Orlando, Florida, this assumption may be wrong. In six dogs that were treated with an AAV-based gene therapy for the blood-clotting disorder hemophilia B, all of them carried the therapeutic DNA within their genomes, and in some cases, the genetic material had inserted near genes known to play a role in cell growth, Science reports.

News: NJIT to Launch New Programs in Cell and Gene Therapy
New Jersey Institute of Technology (NJIT) in collaboration with New Jersey Innovation Institute (NJII), an NJIT corporation, has announced it will soon offer both a Professional Science Masters (PSM) degree program and professional graduate certificate in the rapidly expanding field of cell and gene therapy. The 30-credit M.Sc. degree — available in spring 2020 as part of NJIT’s Department of Chemistry and Environmental Science’s pharmaceutical chemistry master's program — aims to "train the next generation of scientists and engineers in modern aspects of cell and gene therapy, emphasizing cell biology and cellular therapeutic development, regenerative medicine, process development and biomanufacturing, analytical technologies, quality and regulatory compliance.”

Conference: Tri-Conference 2020
The past few years have seen some remarkable successes for gene replacement therapy in diseases, including hereditary blindness, sickle-cell disease, and muscular dystrophy. With the promise of genome editing entering the clinic, there is palpable excitement for the future of genetic therapy. The Inaugural Renaissance of Gene Therapy and Genome Editing conference program at the 27th International Molecular Medicine Tri-Conference, 2-3 March 2020, San Francisco, will showcase topics spanning fundamental research, genome editing (CRISPR and zinc-finger nucleases), preclinical and clinical investigation, as well as considering manufacturing, delivery, and ethical challenges. Hear from leaders in the field of gene therapy and genome editing about the latest advances, future hurdles, and learn where the current-state-of-the art is for changing the course of disease in our time.
Register now...

Conference: Gene Therapy for Blood Disorders
With an approved gene therapy treatment for β-thalassemia in the EU, and hemophilia candidates seeking 2020 approval in the USA and beyond, pioneers in the space must overcome hurdles they have never previously encountered to transform their clinical candidates into a commercial reality. Gene Therapy for Blood Disorders, 3-5 March 2020, Boston, will unite industry leaders across the blood disease space to delve into the specific clinical and commercial challenges faced by companies developing gene therapies for hemophilia, sickle cell disease and β-thalassemia.
Evaluate the best strategies for a commercial launch in blood disease, optimize your trial design, gain insights on revolutionary novel technologies, and ultimately enhance your current hematology gene therapy program to bring transformative treatments to patients more quickly and efficiently.

Register now...

Conference: Inborn Errors of Metabolism Drug Development
Join over 80 inherited metabolic disease drug developers and KOLs to explore how the key challenges prohibiting accelerated drug development are being addressed. The inaugural Inborn Errors of Metabolism Drug Development Summit, 10-12 March 2020, Boston, will provide a discursive platform to examine where pioneering gene therapy modalities intersect with current therapeutic options and explore how diseases can be better targeted in order to address the huge unmet medical need for rare metabolic diseases. This is your comprehensive guide to define your drug development path for 2020. Don’t miss out on your chance to network with industry leaders and be a part of the only enriched industry forum focused on accelerating the development of better effective treatments for inborn errors of metabolism.
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Conference: 10th Spring Meeting ISBioTech
Join ISBioTech for this world renowned 10th Spring Meeting, 23-25 March 2020, Norfolk Virginia covering the development and production of viruses needed for gene and cell therapies as well as human and animal vaccines, development of cellular therapies, plus products developed with baculovirus technology. This is a meeting with real data and methods presented by the individuals and organizations you want to learn from.
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Conference: Innate Killer Summit 2020
Looking at Improving Patient Care by Advancing Innate Understanding & Enhancing Innate Cell Therapies? The Innate Killer Summit Community is here to help with a meeting (24-26 March 2020, San Diego) specifically designed to help you with all your translatability challenges. Accelerate your innate cell immunotherapies to market today.
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Conference: Bioprocessing Summit Europe
The fastest growing bioprocessing meeting, 24-26 March 2020, in Europe is returning in 2020, this time in Barcelona with more ground-breaking science, technologies and exhibitors than ever before! The 8 track conference, which doubled in attendance in 2019, brings together leading bioprocessing scientists to discuss the latest developments and trends in cell culture, cell line, protein expression, recovery, purification, cell and gene therapy manufacturing, and new for 2020, analytical characterisation, formulation and aggregates.
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Conference: 4th Annual Gene Therapy for Rare Disorders Summit
The 4th Annual Gene Therapy for Rare Disorders, 30 March - 2 April 2020, Boston, will focus exclusively on overcoming the late-stage commercial challenges drug developers face when delivering gene therapies to market. With approvals in a number of indications over the last year, and more rapidly approaching on the horizon, the 2020 meeting will leverage the experience from those first to market pioneers, as well as unveiling the strategies the next generation of drug developers are employing to improve efficacy, safety and commercial viability.
Incorporating insights from 60+ industry-leading speakers, this 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 600+ of your colleagues to accelerate the progress of the next generation of gene therapies. This is your comprehensive guide to define your commercial path forward in 2020.

Register now...

- 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
- 10 - 12 March 2020, Inborn Errors of Metabolism Drug Development, 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
- 29 - 30 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
- 3 - 4 June 2020, 2nd Annual Gene and Cell Therapy: Quality Developments to Commercialization Summit, Munich, Germany
- 4 June 2020, Cell & Gene Therapy Strategy 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
- 6 – 7 October 2020, Cell Series UK 2020 - Cell & Gene Therapy Manufacturing, London, UK
- 20 – 23 October 2020, XXVIII Congress of the European Society of Gene and Cell Therapy (ESGCT), Edinburgh UK


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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