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Newsletter September 2020

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News: Study Confirms Efficacy of Gene Therapy for Spinal Muscular Atrophy in Children
In May 2019, the U.S. Food and Drug Administration (FDA) approved a gene replacement therapy for the inherited, progressive neuromuscular disease 5q-linked spinal muscular atrophy (SMA). Approval included all children with SMA under the age of two years; however, the gene therapy had only been studied in children aged up to 8 months. Now, a new study discusses safety and early outcomes in a large cohort of SMA patients under the age of two years who were treated with gene therapy. The report is published in the journal Pediatrics.
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News: New HIV Gene Therapy, CAR-T Treatments Could be on the Horizon for Patients
The National Institutes of Health (NIH) has backed researchers at the University of Southern California and the Fred Hutchison Cancer Center with a five-year, $14.6 million grant to develop a gene therapy that could potentially control HIV without the need for daily medications. Most HIV patients take a well-regimented cocktail of medications each day to control the virus. This therapy could change that.
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Conference: 3rd Cell Therapies and Immunotherapy Conference
Organized by the Society for Biological Engineering, this online event, 1-3 October 2020, brings an international assembly of experts to discuss new research in the areas of cancer immunology, clinical applications, CAR T cells, synthetic & systems biology, checkpoint Inhibition, oncolytic viruses and tumor microenvironment engineering. Plan today to join us in our examination of new discoveries that will ultimately change the future of healthcare. Visit the website of the 3rd Cell Therapies and Immunotherapy Conference to learn more.
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Conference: Cell Therapy Manufacturing & Gene Therapy Congress EU
In light of the current global climate, it is more important than ever for the cell & gene therapy community to connect with one another, strategize and share ideas – All in a safe, responsible way. With our dedication to address the most pressing issues, our cell and gene therapy events, now taking place 19-22 October, have undergone a timely transformation, moving to an all-digital format and offering attendees the ability to participate in virtual content, exhibits and networking opportunities.
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Conference: TCR-based Therapies Summit
The only dedicated end-to-end, industry led TCR therapy summit. 26-28 October 2020 is a unique opportunity to delve into the details of TCR development. This technical meeting is a rare opportunity to learn from 20+ industry experts from Ziopharm, Immatics US, TScan Therapeutics and many more. Gain insights on their various approaches and be the first to hear cutting-edge data from this fast-paced industry.
Covering the most exciting developments and promising clinical data, the online TCR-based Therapies Summit will propel your research ahead in 2020 with optimized target identification, supercharged T-cell modifications and insights to the future of manufacturing. For leaders in the TCR space, this is not a meeting to be missed! Gene Therapy Net member can use code GTN10 upon registration for 10% off.

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Conference: Patient-to-Patient Supply for Cell Immunotherapies Summit
As an increasing number of candidates enter the clinic and promise to be a mainstay in the future of cancer treatment, cell therapies pose unique supply chain challenges. From time and temperature sensitivity, to patient-facing services and integrated platforms, a seamless vein-to-vein supply chain is central in maintaining cell immunotherapy safety and efficacy at scale and maximizing the patient experience.
From 29-30 October 2020 join industry experts from large pharma, biotech, standards agencies and supply chain and logistics service providers at the Patient-to-Patient Supply for Cell Immunotherapies Summit. Hear 16+ real-world case studies led by supply chain, strategic sourcing, apheresis, and technical operations experts from top cell therapy providers. Ensure effective forecasting, risk management, quality assurance and service at scale to meet clinical to commercial demand.
Build, adapt and coordinate the vein-to-vein supply of life-changing cell therapies, virtually network and establish yourself in the cell therapy supply chain community, and share your own experiences to help develop best practice in vein-to-vein cell therapy supply.

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Conference: Reuters Cell & Gene Therapy USA event
With 9 cell and gene treatments on the market, 362 in the pipeline, and US industrial policy accelerating pathways to market, the race to commercialize – getting these incredible solutions to patients - is well and truly on. Is your organization ready to speed your therapy to market.
Reuters Events' brand new, free to attend online event, Cell & Gene Therapy USA 2020, 2 November 2020, will bring together 2000+ cell and gene stakeholders including leaders in pharma, biotech, payer organizations, patients and CMOs to tackle patient identification, payer, HCP and patient education and engagement, manufacturing, access and affordability, bioethics, internal alignment and much, much more.

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Conference: 6th Annual Biopharma Development and Production (BDP) Week 2020
The 6th Annual Biopharma Development and Production Week 2020, 3-4 November 2020, is your go-to event to reach out to key decision makers in the pharmaceutical industry. The event will address the 2020 investment climate, technological advancements, industry case studies, and partnerships and expansion opportunities across key value chains. This umbrella event spans 3 co-located conferences – 10th Annual Biomanufacturing, 9th Annual Cell Line Development & Engineering Asia and 2nd Annual Cell & Gene Therapy Manufacturing.
With over 100 local and global expert speakers and 50 case studies, this year’s conference is expected to be much more popular with China leading as the 2nd largest biologics market in the world. The Chinese industry approaches a tipping point as companies re-evaluate current trends and strategies to achieve commercial excellence with numerous shifts on the horizon.

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Conferences
- 15 - 17 September 2020, Bioanalytical strategies for Cell & Gene Therapies workshop, Lisbon, Portugal
- 21 - 22 September 2020, Bioproduction Summit on Advances in AAV for Gene Therapy & Protein Production, San Francisco, CA
- 22 - 24 September 2020, Advanced Therapies Manufacturing Strategy Summit, Online event
- 22 - 25 September 2020, Patient Centric Cell Therapy Supply Chain Summit, Boston, MA
- 23 - 25 September 2020, American Society of Gene & Cell Therapy (ASGCT) Policy Summit 2020, Online event
- 30 September 2020, Gamma Delta T Therapies Summit, Online event
- 1 – 3 October 2020, 2020 Cell Therapies and Immunotherapy Conference, Online event
- 6 – 7 October 2020, Cell Series UK 2020 - 6th Annual Cell & Gene Therapy Manufacturing Congress, Online event
- 15 - 16 October 2020, Global Cell and Gene Therapy Innovation Leaders Shanghai Forum - Asia Pharma R&D Leaders Summit 2020, Shanghai, China
- 20 – 21 October 2020, Cell & Gene Therapy Research & Development Congress Asia 2020, Taipei, Taiwan
- 26 - 27 October 2020, 2nd Annual Gene and Cell Therapy: Quality Developments to Commercialization Summit, Munich, Germany
- 26 - 28 October 2020, Allogeneic Cell Therapies Summit 2020, Boston, MA
- 26 - 28 October 2020, 4th Gene Therapy for Rare Disorders Europe, London, UK
- 26 - 28 October 2020, TCR-based Therapies Summit, Online event
- 29 - 30 October 2020, Patient-to-Patient Supply for Cell Immunotherapies Summit, Online event
- 2 November 2020, Cell & Gene Therapy USA 2020 event, Online event
- 3 - 4 November 2020, 6th Annual Biopharma Development and Production (BDP) Week - 2nd Annual Cell and Gene Therapy Manufacturing, Shanghai Hongqiao, China
- 4 - 6 November 2020, 6th Annual Genome Editing congress, Online event
- 23 - 24 November 2020, Ophthalmic Drugs Conference - Gene Therapies, London, UK
- 30 November - 3 December 2020, 2nd Annual Gene Therapy Analytical Development, Philadelphia, PA
- 1 - 3 December 2020, Cellular Immunotherapies for Solid Tumors Digital Summit, Online event
- 1 - 3 December 2020, 3rd Neuromuscular Drug Development Summit - Gene Therapies, Online event
- 8 - 10 December 2020, Gene Therapy for Neurological Disorders Summit, Boston, MA
- 8 - 10 December 2020, 3rd RNA-Targeted Drug Discovery Summit, Online event

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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, Zika Virus Net and Corona Virus Net.
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