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Newsletter January 2021

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News: Liver tumor in gene therapy recipient raises concerns about virus widely used in treatment
It’s troubling news that gene therapy researchers have long anticipated: A hemophilia patient injected with a virus carrying a therapeutic gene in a clinical trial has developed a liver tumor. The U.S. Food and Drug Administration (FDA) has halted the associated clinical trials, and uniQure, the Dutch firm behind the studies, is now investigating whether the virus itself caused the cancer. Gene therapy experts say that’s unlikely. The patient had underlying conditions that predisposed him to liver cancer. Still, scientists say it’s crucial to rule out any role for adeno-associated virus (AAV), the viral delivery system, or vector, that is used in hundreds of other gene therapy trials. “Everyone will want to know what happened,” says physician-scientist David Lillicrap of Queen’s University, a hemophilia researcher who was not involved with the uniQure study.
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Conference: Global Onco Cell Therapy Virtual Summit
The Onco Cell Therapy Series kicks off 2021 with its most interactive event ever, with 20+ panel conversations, townhall meetings, roundtable sessions and coffee break style networking.
The Global Onco Cell Therapy Virtual Summit, January 20-21, 2021, provides a platform for information sharing to enable the discovery, translation and commercialisation of cell therapies in oncology by connecting senior representatives in pharma, biotechs and academia. The summit will address the research, regulatory, logistical and technical challenges that are holding back the field.
Whilst bringing together the key decision makers for oncological cell therapies, this year’s summit will have an added focus on the data releases and mergers that are driving the surge of interest in harnessing alternative cell therapies in cancer treatments.
All tickets include a FREE access to a pre-event NK Day and are 2 tickets for the price of 1 on standard rates. Quote ‘2FOR1’ when booking online. Gene Therapy Net readers also receive an additional 10% discount!! Quote GTN10 to claim.

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Conference: Bioprocessing Summit Europe Virtual
The Bioprocessing Summit Europe, March 16-17, 2021, brings together leading bioprocessing scientists to share day-to-day challenges and practical solutions for the development, manufacture and quality of antibodies, vaccines and gene therapies.
This four-track virtual conference, which is the fastest growing bioprocessing conference in Europe, presents detailed case studies and interactive discussions in cell culture, scale-up, continuous processing, recovery, purification, process and product characterization, and formulation.

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Conference: Innate Killer Summit 2021
Advance Gene Engineering, Persistence, and Scalable Expansion of NK, Gamma-Delta T, and NKT Cell Therapies to Develop Cost-Effective Allogeneic Treatments for Liquid and Solid Tumors.
2020 has been a game-changing year for the NK cell community. Positive clinical results have forced the field to wake up to the potential of these pioneering approaches. However, this increased clinical understanding highlights the challenges in producing long-lasting, scalable products.
The Innate Killer Summit is returning for its 6th year digitally (March 23-25, 2021), to improve Patient Care by Advancing Innate Understanding & Enhancing Innate Cell Therapies.
200+ experts from the likes of FDA, Nkarta, Cytovia and AgenTus to hear the unpublished clinical data and innovative manufacturing processes to accelerate the clinical and commercial development of innate cell therapies. Become part of the Innate Killer Community to discover how to target solid tumor indications with off-the-shelf innate cell therapies!
With over 40 speakers sharing their expertise, and the NK field exploding with new companies, investment, and clinical collaborations, it is more important than ever to stay ahead of the curve.

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Conference: Gene Therapy Patient Engagement
Gene Therapy Patient Engagement, March 23-25, 2021, will delve into the specific details of how to integrate the patient voice at every stage of gene therapy development, enabling you to collaborate with the patient community more effectively than ever before.
By leveraging knowledge and experience from patient advocacy leaders at gene therapy companies, patient advocacy organizations and patients themselves, this digital meeting will probe a level beyond broad concepts and explore case studies on defining novel outcomes for gene therapy trials, managing the unique challenges involved in long-term follow-ups and when, how and with which tools to engage patients most effectively

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Conference: Gene Therapy Comparability Summit
Pioneer effective CMC & analytical comparability studies to maintain safety, quality & efficacy at every stage of manufacturing. The Gene Therapy Comparability Summit , March 30 - April 1, 2021, Gene Therapy Comparability Summit will incorporate insights from leading CMC, Regulatory, Analytical and Process Development experts on challenges like ensuring manufacturing processes are comparable when changing facilities and cell lines, developing effective bioassays for use in comparability studies and handling the limited gene therapy lots available.
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Conferences
- 19 - 21 January 2021, PepTalk 2021 Conference, Online event
- 20 - 21 January 2021, Global Onco Cell Therapy Summit, Online event
- 26 - 28 January 2021, Inaugural TGFβ for Immuno-Oncology Drug Development Summit - Gene Therapies, Online event
- 9 - 11 February 2021, Gene Therapy Medical Affairs Summit, Online event
- 10 - 11 February 2021, RNA Therapeutics 2021, Online event
- 16 - 18 February 2021, 28th Annual Molecular Med Tri-Con Virtual Conference & Expo, Online event
- 16 - 18 February 2021, CAR-TCR Digital Summit Europe, Online event
- 22 - 25 February 2021, 4th Annual Gene Therapy for Rare Disorders, Boston, MA
- 23 February 2021, Cell & Gene Therapy Day, Online event
- 23 - 25 February 2021, 9th Neurodegenerative Drug Development Summit, Online event
- 23 - 26 February 2021, IO360° Summit - Gene Therapies, Online event
- 9 - 11 March 2021, 2nd Gene Therapy for Blood Disorders meeting, Online event
- 15 - 17 March 2021, International Society for BioProcess Technology 10th Spring Meeting - Viral vectors and vaccines, Norfolk, Virginia
- 16 - 17 March 2021, 4th Annual Bioprocessing Summit Europe Virtual, Online event
- 23 March 2021, Macrophage Therapy Symposium, Online event
- 23 - 25 March 2021, Innate Killer Summit 2021, Online event
- 23 - 25 March 2021, Gene Therapy Patient Engagement, Online event
- 30 March - 1 April 2021, Gene Therapy Comparability, Online event
- 14 - 16 April 2021, World Smart Bioprocessing: Pharma 4.0 Summit, Berlin, Germany
- 20 - 21 April 2021, Formulation & Delivery Europe: Gene and Cell Therapies, Online event
- 11 - 14 May 2021, American Society of Gene and Cell Therapy (ASGCT) 24th Annual Meeting, Online event
- 26 - 28 May 2021, International Society for Cell and Gene Therapy (ISCT) New Orleans virtual meeting, Online event
- 14 - 18 June 2021, 9th International Symposium on Monolith - DNA/RNA, vaccines, gene therapy vectors, Portoroz, Slovenia
- 29 - 30 June 2021, Formulation & Delivery US: Gene and Cell Therapies, San Diego, CA
- 31 August - 2 September 2021, CAR-TCR Summit USA, Boston, MA
- 21 - 22 September 2021, Formulation & Delivery UK: Gene and Cell Therapies, London, UK
- 22 - 23 September 2021, Oligonucleotide Therapeutics and Delivery conference, London, UK
- 12 - 13 October 2021, Pharma Europe 2021 - Cell & Gene Therapies, Barcelona, Spain
- 18 - 20 October 2021, Advancing Gene Therapy 2021, Boston, MA
- 19 - 21 October 2021, XXVIII Congress of the European Society of Gene and Cell Therapy (ESGCT), Brussels, Belgium
- 10 - 11 November 2021, Pharma Manufacturing: Gene and Cell Therapies, Online event
- 22 - 23 November 2021, Ophthalmic Drugs Conference - Gene Therapies, London, UK

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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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