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Newsletter July 2021

News: A year after getting UniQure's gene therapy, hemophilia patients are still doing better
One year after receiving an experimental gene therapy developed by the Dutch drugmaker UniQure, patients with hemophilia B aren't having nearly as many bleeding issues as they used to have. In hemophilia, genetic mutations prevent the body from making proteins needed to clot blood. People with the less common, "B" form of the disorder are missing a protein known as Factor IX. UniQure's medicine is meant to provide a working version of the gene so patients can generate their own clotting protein, and rely less — or ideally, not at all — on so-called replacement factor treatments.

Webinar: Cell and gene data innovation and multi-stakeholder data transfer
Utilize data flow in cell and gene therapies from R&D to launch to win the marketplace with science and data. Learn how here: Cell and gene data innovation and multi-stakeholder data transfer, July 29, 2021.
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Conference: World Orphan Drug Congress USA In-person
Join largest rare disease and orphan drug conference, globally, #WODC, this August 25-27, 2021 in Oxon Hill, MD. Save 20% on your pass with code MPN20.
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Conference: 6th CAR-TCR Summit
Harnessing Disruptive CAR & TCR Based Cell Technologies to Supercharge Safe, Effective & Globally Accessible Cell Immunotherapies in Oncology & Beyond. The 6th Annual CAR-TCR Summit returns in 2021, August 30-September 2, 2021, as the industry-leading comprehensive forum with one goal: to engineer a disease-free world
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Conference: Next Generation Cancer Vaccine Development Summit
This September 7-9, 2021 industry experts will meet to discuss the cutting-edge in Antigen Optimization, Vaccine Platforms, Transcriptional Availability and more at Next Generation Cancer Vaccine Development Summit. The digital event will focus on overcoming the translational and clinical challenges of developing cancer vaccines, including finding the right antigens, reassessing patient populations, understanding immune suppression and resistance patterns, and exploring case studies covering Ovarian Cancer, Pancreatic Cancer, Glioblastoma, Lung Cancer, Melanoma, Malignant pleural mesothelioma (MPM), Prostate Cancer, and Advanced Solid Tumors
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Conference: Gene Therapy for Metabolic Disorders
Gene Therapy for Metabolic Disorders, September 14-16, 2021, will unite large pharma and innovative biotechs to collectively overcome translational drug development barriers to deliver on the transformative promise of gene therapy approaches for patients in need.
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Conference: 2nd Gene Therapy for Ophthalmic Disorders
Returning in September 21-23, 2021 as a digital event, the 2nd Gene Therapy for Ophthalmic Disorders meeting will target the industry’s translational challenges in targeting both rare and common ophthalmic disorders including wet AMD, dry AMD, DME, IRDs and more.
Discover novel vectors and administration routes for optimal retinal delivery with 30+ world class speakers from the likes of GenSight Biologics, Biogen, Gyroscope Therapeutics, Novartis, Horama and REGENXBIO Join 150+ fellow industry attendees from discovery, preclinical and clinical backgrounds, to learn lessons in rare disorders and seize the opportunity in common ophthalmic disorders.

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Conference: Oligonucleotide Therapeutics and Delivery conference
Oligonucleotide therapeutics - the emerging medicine class - are harnessing the therapeutic benefit of targeting genetic material via antisense, mRNA, RNAi, saRNA and siRNA. SMi’s inaugural Oligonucleotide Therapeutics and Delivery conference, September 22-23, 2021 in London, is here to keep you updated. By bringing together leading representatives of pharmaceutical, biotechnology and academic institutions, the 2-day conference will provide first-hand information on the latest clinical trial candidates and a platform for exchanging ideas for tackling the biggest challenge: DELIVERY. From optimizing particle size to choosing the right nanocarrier systems, the conference will address delivery to non-hepatocyte cells such as cancerous tissues and overview the most successful platforms. Discussions will be centered around improving targeted therapy uniting the field’s biggest players to share their opinions.
Established on the success of our RNA Therapeutics series, we look forward to welcoming you at Oligonucleotide Therapeutics and Delivery to join the conversation around maximizing the potential of oligo-based treatments.

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- 19 - 21 July 2021, 2nd Gene Therapy for Neurological Disorders Europe, Online event
- 27 - 29 July 2021, Gamma Delta T Therapies Summit, Online event
- 3 - 5 August 2021, 2nd Gene Therapy Immunogenicity Summit, Online event
- 16 - 19 August 2021, 13th Annual The Bioprocessing Summit, Boston, MA
- 25 - 27 August 2021, World Orphan Drug Congress USA, Maryland, USA
- 30 August - 2 September 2021, 6th CAR-TCR Summit, Online event
- 7 - 9 September 2021, Next Generation Cancer Vaccine Development Summit, Online event
- 14 - 16 September 2021, Gene Therapy for Metabolic Disorders Summit, Online event
- 21 - 22 September 2021, Formulation & Delivery UK: Gene and Cell Therapies, London, UK
- 21 - 23 September 2021, 2nd Gene Therapy for Ophthalmic Disorders, Online event
- 22 - 23 September 2021, Oligonucleotide Therapeutics and Delivery conference, London, UK
- 28 - 29 September 2021, Cell & Gene Therapy USA - Reuters Event, Online event
- 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, 2nd Annual Dry AMD Therapeutic Development Summit, Online event
- 19 - 21 October 2021, XXVIII Congress of the European Society of Gene and Cell Therapy (ESGCT), Online event
- 27 - 29 October 2021, World Smart Bioprocessing: Pharma 4.0 Summit, Berlin, Germany
- 8 - 10 November 2021, 2nd Annual Inborn Error of Metabolism Drug Development Summit - Gene Therapies, Boston, MA
- 10 - 11 November 2021, Pharma Manufacturing: Gene and Cell Therapies, Online event
- 16 - 17 November 2021, Onco Cell Therapy Summit (OCTS) USA 2021, Boston, MA
- 22 - 23 November 2021, Ophthalmic Drugs Conference - Gene Therapies, London, UK


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