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Newsletter August 2018

News: FDA Seeks to Enhance Manufacturing of Cell and Gene Therapies
More consistent and reliable production processes are critical for advancing innovative treatments. While cell- and gene-therapy products hold the promise of transforming the treatment of many diseases, difficulties in achieving consistent process control could stymie advancement in this field, cautions Peter Marks, director of FDA’s Center for Biologics Evaluation and Research (CBER). It’s “not simple” to bring a clinical production process to commercial scale, he says, voicing fears that important manufacturing issues could hinder efforts to advance new discoveries able to transform medical care.

News: New EU initiatives to optimize the interplay between the Pharma and the GMO framework
The EU's Regulation on advanced therapies, is designed to ensure the free movement of advanced therapy products within Europe, to facilitate access to the EU market, and to foster the competitiveness of European companies in the field, while guaranteeing the highest level of health protection for patients. A Good Practice document on the assessment of GMO-related aspects in the context of clinical trials with human cells genetically modified has been developed by the national competent authorities and the Commission services.

News: Caution required for using CRISPR/Cas9 in potential gene therapies
Scientists at the Wellcome Sanger Institute have discovered that CRISPR/Cas9 gene editing can cause greater genetic damage in cells than was previously thought. These results create safety implications for gene therapies using CRISPR/Cas9 in the future as the unexpected damage could lead to dangerous changes in some cells. Reported in the journal Nature Biotechnology, the study also revealed that standard tests for detecting DNA changes miss finding this genetic damage, and that caution and specific testing will be required for any potential gene therapies.

News: First cohort enrolled in trial with innovative gene therapy for arthritis
Arthrogen, a clinical stage gene therapy company together with the Centre for Human Drug Research (CHDR), a Leiden based independent clinical research organization, announced that the first three arthritis patients have been enrolled in a phase Ib gene therapy trial for treatment of arthritis with ART-I02. ART-I02 is an adeno-associated virus (AAV5) vector encoding the human IFN-β gene under control of an inflammation-responsive promoter. ART-I02 is designed to produce the anti-inflammatory protein IFN-β in the synovial cells in the joint. The aim is to achieve a sustained clinical remission with a single treatment. This current clinical phase Ib trial, evaluates the safety, tolerability, pharmacokinetics, immunogenicity and anti-inflammatory activity of ART-I02 treatment in patients with rheumatoid arthritis (RA) or osteoarthritis (OA). In total 12 patients with RA or OA in their hand joints will be included. Parallel to this study, a similar phase Ib trial has started in Canada treating 15 patients with RA in their wrist.

Conference: International Conference on Cell and Gene Therapy 2018
The International Conference on Cell and Gene Therapy 2018 (September, 10-11, Dublin) deals with the diagnosis, prevention, and treatment & therapies of diseases of the gene specific, cell specific, organ specific and including the latest techniques in current era. Cell and Gene Therapy 2018 is an extraordinary event designed for International medical health professionals and Immunologist, Oncologist, Neurologist, Genetic researchers, many organizations and associated people of gene and cell research to facilitate the dissemination and application of research findings related to Cell and Gene.The conference invites participants from all leading universities, clinical research institutions and diagnostic companies to share their research experiences on all aspects of this rapidly expanding field and thereby, providing a showcase of the latest techniques.

Conference: International Conference on Lymphocyte Engineering
The International Conference on Lymphocyte Engineering, ICLE 2018 (September, 13-15, Madrid) will provide a dedicated platform to advance research, clinical translation and treatment in immuno-gene therapy. ICLE brings together immunotherapy with cell and gene therapy to foster interdisciplinary collaborations, deepening the connection between basic and clinical research across related disease specialties: oncology, pathogenic diseases, autoimmunity, immunodeficiencies and others.

Conference: Orphan Drugs and Rare Diseases Conference
The Orphan Drugs and Rare Diseases Conference (October 17 – 18, London, UK) will gather industry experts and address the opportunities and challenges within the rare disease treatment field. Programme covers such topics as: pricing and reimbursement of orphan drugs; challenges of patient recruitment and patient-centric research; MHRA’s perspective on benefit-risk assessment in rare diseases; clinical development of orphan drugs for rare disease plus many others.

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Conference: Gene Therapy for Rare Disorders Europe
The Gene Therapy for Rare Disorders Europe meeting (October 22-24, London, UK) is the uniquely focused conference uniting industry leaders to discuss the late-stage challenges that need to be overcome to deliver more gene therapies to market. Rather than early stage basic science, this meeting is solely devoted to addressing the latest clinical, manufacturing, regulatory and commercialization challenges facing this rapidly evolving field. This is a comprehensive guide to define your commercial path forward.

Gene Therapy Net newsletter readers are eligible for a special 20% discount to attend this meeting. If you would like to attend at the discounted rate, please email This email address is being protected from spambots. You need JavaScript enabled to view it. and quote VIP Code 9709GTN


Conference: Annual Congress on CRISPR-Cas9 Technology
The Annual Congress on CRISPR-Cas9 Technology (October 24-25, Boston, USA) is a Research-scientific knowledge bridge, that aims bring together multi-disciplinary luminaries for Thriving innovation in the Biotechnology. The scientific conferences have been carefully structured so as to share knowledge and thoughts through presentations and exhibitions. CRISPR-2018 event with sessions covers all aspects of biotech-driven technique CRISPR and addresses the key issues currently affecting its researches. Attendees can look forward to hearing about the different strategies taken to improve ongoing research and decipher how to overcome technical limitations in research development. This conference is where pharm, investors and Life Science companies find partners, access innovation, find funding and brainstorm the solutions to further their business needs.

Gene Therapy Net members will be provided 30% discount, and they can register through our discount portal


Conference: 2nd Annual Summit on Stem Cell Research, Cell & Gene Therapy
The theme of the 2nd Annual Summit on Stem Cell Research, Cell & Gene Therapy (November 9-10, Atlanta, USA) is "Bridging the gap from Basic Cell Science to Advanced Cellular Therapies for a Better Life" which provides a unique way to present the evidences on latest advancements and researches with a holistic approach to different areas of interest. The conference tracks are designed in such a way so as to cover all the areas of researches involved with Cell Therapy and Stem Cell Research and their impacts on human health. The main aim of this conference is to provide a platform to all the scientists, doctors, medical professionals and business delegates who will undoubtedly enhance the conference by giving their views regarding recent breakthroughs to all the attendees.

- 13 - 17 August 2018, Bioprocessing Summit 2018 - Gene Therapy, Boston, MA
- 20 - 21 August 2018, EuroSciCon Conference on Cell and Gene Therapy, Amsterdam, The Netherlands
- 4 - 7 September 2018, 4th Annual CAR-TCR Summit 2018, Boston, MA
- 10 - 11 September 2018, International Conference on Cell and Gene Therapy, Dublin, Ireland
- 13 - 15 September 2018, First International Conference on Lymphocyte Engineering (ICLE 2018), Madrid, Spain
- 2 – 3 October 2018, Advanced Therapies Commercialisation Congress, Amsterdam, The Netherlands
- 10 – 11 October 2018, Cell & Gene Therapy: From manufacturing to patient access, London, UK
- 15 – 16 October 2018, Rare Diseases and Orphan Products Breakthrough Summit, Washington, DC
- 16 – 19 October 2018, XXVI Congress of the European Society of Gene and Cell Therapy (ESGCT), Lausanne, Switzerland
- 17 – 18 October 2018, Orphan Drugs and Rare Diseases Conference, London, UK
- 22 – 24 October 2018, Gene Therapy for Rare Disorders Europe, London, UK
- 22 – 24 October 2018, Precision CRISPR Drug Development and Gene Therapy Congress, London, UK
- 24 – 25 October 2018, Annual Congress on CRISPR-Cas9 Technology, Boston, MA
- 25 – 26 October 2018, 4th Annual Cell & Gene Therapy Congress, London, UK
- 6-8 November 2018, World Orphan Drug Congress 2018, Barcelona, Spain
- 8-9 November 2018, 4th Annual Genome Editing UK Congress, London, UK
- 9-10 November 2018, 2nd Annual Summit on Cell Therapy and Stem Cell Research 2018 Conference, Atlanta, GA
- 23 November 2018, British Society for Gene and Cell Therapy (BSGCT) Annual Conference Autumn 2018, London, UK
- 30 January - 1 February 2019, Gene Therapy – Ready for the Market?, Frankfurt am Main, Germany
- 21 - 22 March 2019, 9th International Conference and Exhibition on Advanced Cell and Gene Therapy, Rome, Italy
- 3 – 5 April 2019, 4th Spring school of the European Society of Gene and Cell Therapy (ESGCT), Naples, Italy
- 29 April - 2 May 2019, American Society of Gene and Cell Therapy (ASGCT) 22th Annual Meeting, Washington, DC
- 21 – 25 October 2019, XXVII Congress of the European Society of Gene and Cell Therapy (ESGCT), Barcelona, Spain


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