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Newsletter October 2020

News: Gene Therapy in Mesothelioma Patients
Around 1 in 16 people will be diagnosed with lung cancer in their lifetime; this accounts for about 13% of all new cancer diagnoses. However, even with a relatively lower diagnosis rate, lung cancer currently has the highest number of cancer deaths at nearly 22%. Among all the various subtypes of cancer categorized under the umbrella term, “lung cancer,” mesothelioma is one of the most lethal forms. Despite accounting for around .2% of all cancer diagnoses in the United States, mesothelioma’s lethality is among the highest of all forms of cancer. Pleural mesothelioma, the most common form of this cancer, has a 10-year survival rate of only about 5%. Currently, the only known cause of mesothelioma is asbestos exposure, and although no amount of exposure is safe, it is important to note that your risk of developing mesothelioma drastically increases the longer you are exposed. In addition, due to a prolonged latency period, many patients are diagnosed far too late for proper treatment — usually around the age of 65-74.

News: FDA grants orphan drug designation for LHON gene therapy
Neurophth Therapeutics has received orphan drug designation from the FDA for NR082 for the treatment of Leber hereditary optic neuropathy associated with ND4 mutation, according to a press release. Between 70% and 90% of LHON cases are caused by ND4 mutation, according to the release.

Conference: Next Generation CAR & T Cell Therapies
Next Generation CAR & T Cell Therapies, November 2-5, 2020, is the only CAR & T Cell Therapies focused event on the West Coast. Pioneering point of care with innovative manufacturing and commercialization strategies. Revolutionizing cell and gene engineering - ground breaking autologous and allogeneic treatments for blood borne and solid tumors.
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Conference: 6th Annual Genome Editing Congress
The 6th iteration of BDP Week, November 3-5, 2020, will address the 2020 investment climate, technological advancements, industry case studies, and partnerships and expansion opportunities across key value chains. With improved networking windows and elevated exhibition platforms, join us as the industry convenes at this highly anticipated annual gathering of biopharma leaders.
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Conference: 2nd Annual Gene Therapy Analytical Development
In the context of rapid gene therapy progress and mounting regulatory scrutiny, the 2nd Annual Gene Therapy Analytical Development Summit, November 30 – December 3, 2020, will virtually unite large pharma and innovative biotechs to discuss the full and comprehensive range of analytical tools required to guarantee the consistency, quality and safety of gene therapy products.
Focused specifically on addressing the unique analytical challenges posed by gene therapy vectors, this technical scientific conference will unite the Analytical Development, QC, CMC, and Process Development leaders grappling with these challenges first-hand.
Incorporating tracks taking a deep dive into bioassays, molecular biology, and analytical chemistry, this is your opportunity to gain extensive technical information enabling you to solve the exact challenges you’re encountering. Enhance your existing analytical methods and explore innovative novel tools to support safe and effective gene therapy development.

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Conference: Cellular Immunotherapies for Solid Tumors Digital Summit
The cell therapy revolution has changed the face of cancer treatment proving a curative success in liquid tumors. Still, with 90% of all cancers arising from solid tumors and extremely little efficacy shown, the time has come to invest in R&D to tackle this huge unmet need and take advantage of this vast untapped market. Join the race for this next frontier for cell immunotherapy, capitalize on the huge commercial opportunity, and confront this great unmet need with the first clinically safe and effective solid tumor therapy.
The Cellular Immunotherapies for Solid Tumors Summit, December 1-3, 2020, is the first digital forum to host industry and academic experts including Immatics, GSK, and the University of Pennsylvania who are pioneering research in the solid tumor space. Unveiling strategies to identify solid tumor targets, enhance cell persistence, and overcome the tumor microenvironment with combination and arming strategies, this focused online forum will provide your team with the learnings needed to progress into the clinic successfully. Use code GENETHERAPY10 for a 10% discount on our registration.

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Conference: 3rd Neuromuscular Drug Development Summit
Virtual Neuromuscular Drug Development Summit, December 1-3, 2020: Develop clinically curative therapeutics for neuromuscular disorders with Biogen, PTC Therapeutics, Avidity Biosciences and more.
This exclusive summit is putting the hottest topics in neuromuscular drug development under the microscope, addressing questions including: validating clinically meaningful endpoints to prove drug efficacy; optimizing your clinical trial design to define the right patient populations, and dosing regimens and evaluate approaches to integrate biomarkers into your programs. This intimate, industry-focused forum will promote cross-stakeholder discussion and networking. Join 60+ of your peers to accelerate the development of clinically efficacious & meaningful therapies for patients in need.

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Conference: 2nd RNA Editing Summit
Korro Bio have just announced that their OPERA platform has secured $91.5 million in funding.OPERA (Oligonucleotide Promoted Editing of RNA), harnesses the body's natural base editing system. Korro's approach relies on nucleotide deamination, an endogenous RNA-modifying process that already happens in cells. It has the potential to repair disease-causing mutations at the RNA level.
Building on the success of last year's meeting, the 2nd RNA Editing Summit, December 1-3, 2020, is a very timely meeting dedicated to pioneering research and therapeutic applications of RNA editing into a clinical and commercial reality.

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Conference: Breaking Through, Delivering on the Promise of Gene Therapy
Breaking Through brings together Canadian and international leaders to explore the past, present, and future of somatic gene therapy research and practice. This two-day event, delivering on the Promise of Gene Therapy, December 2-3, 2020, will examine the successes, challenges and opportunities from the bench to the bedside. It will also feature the CCA Expert Panel on the Approval and Use of Somatic Gene Therapies in Canada, who will discuss the findings and implications of their report, due to be released in November. Join us for this live and interactive two-day event, available in both official languages.
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- 15 - 16 October 2020, Global Cell and Gene Therapy Innovation Leaders Shanghai Forum - Asia Pharma R&D Leaders Summit 2020, Shanghai, China
- 19 – 22 October 2020, Cell Therapy Manufacturing & Gene Therapy Congress EU, Online event
- 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, Online event
- 26 - 28 October 2020, 4th Gene Therapy for Rare Disorders Europe, Online event
- 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
- 2 - 5 November 2020, Next Generation CAR & T Cell Therapies, Online event
- 3 - 5 November 2020, 6th Annual Biopharma Development and Production (BDP) Week - 2nd Annual Cell and Gene Therapy Manufacturing, Online event
- 4 - 6 November 2020, 6th Annual Genome Editing congress, Online event
- 30 November - 3 December 2020, 2nd Annual Gene Therapy Analytical Development, Online event
- 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
- 1 - 3 December 2020, 2nd RNA Editing Summit, Online event
- 2 - 3 December 2020, Breaking Through: Delivering on the Promise of Gene Therapy - Gairdner Ontario International Symposium, Online event
- 8 - 10 December 2020, Gene Therapy for Neurological Disorders Summit, Online event
- 8 - 10 December 2020, 3rd RNA-Targeted Drug Discovery Summit, Online event


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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