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Newsletter June 2019

News: Avexis Receives FDA Approval For Zolgensma, First And Only Gene Therapy For Pediatric Patients With Spinal Muscular Atrophy
AveXis, a Novartis company, today announced the US Food and Drug Administration (FDA) has approved Zolgensma® (onasemnogene abeparvovec-xioi) for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Zolgensma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time intravenous (IV) infusion. Zolgensma is the first and only gene therapy approved by the FDA for the treatment of SMA, including those who are pre-symptomatic at diagnosis.

Site Update: Legislation updates for European countries
The implementation of the European Union (EU) directives and guidelines for clinical gene therapy trials in the different EU member states and other European countries has been updated for the following countries: Austria, Belgium, Bulgaria, Cyprus, Czech Republic, Denmark, Estonia, Finland, France, Germany, Greece, Hungary, Iceland, Ireland, Italy, Latvia, Lithuania, Luxembourg, Malta, Netherlands, Norway, Poland, Portugal, Romania, Slovak Republic, Slovenia, Spain, Sweden, Switzerland, United Kingdom.

The update is based on the EU overview of national regulatory requirements (EU countries and Norway) for medicinal products containing GMOs. This overview has been developed in 2018 as a result of joint efforts to harmonize between the GMO legislation and the legislation on medicinal products.


Conference: NPD – Neuropsychiatric Drug Development - neural gene therapy
Built with thought-leaders at Sage Therapeutics, Janssen and Lundbeck the 2nd Annual Neuropsychiatric Drug Development Summit (NPD), Boston, July 23-25, will unravel the complexities of developing truly clinically transformative neuropsychiatric drugs. Uniting 90+ drug developers from all corners of the world, NPD will be your opportunity to discuss, in depth, the challenges with preclinical models, precision medicine and target selection that have been stifling the progress of your neuropsychiatric drug development. Join your peers at NPD to stride towards developing clinically transformative neuropsychiatric drugs.

Conference: The Bioprocessing Summit on Cell and Gene Therapy
CHI’s Cell and Gene Therapy CMC Analytics and Manufacturing conference, Boston, 12-16 August, is industry’s leading technical meeting for overcoming the latest challenges in CAR T, AAV and viral vector analytics, process development and commercial manufacturing, with two 5-day tracks dedicated to Cell Therapy CMC and Manufacturing AND Gene Therapy CMC and Manufacturing. Key themes across both meetings include product and process characterization, cost-effective scale up, vector quality, regulatory expectations and technical development strategies.

Conference: Blood-Brain Barrier Delivery Summit (B3DD)
The Blood-Brain Barrier Delivery Summit (B3DD), Boston, 13-15 August is the industry’s first conference dedicated to addressing the translational challenges of effective and safe delivery of CNS drug candidates. With an emphasis on biologics, and built with the field's leading minds from the likes of Biogen, Genentech, Armagen & Denali, the B3DDSummit will unite over 60 industry stakeholders giving you the opportunity to develop new partnerships and build on existing relationships to ensure you have the connections to accelerate your drug’s development

- 18 - 20 June 2019, CAR-TCR Summit Asia 2019, Shanghai, China
- 18 - 20 June 2019, Next generation CAR & T Cell Therapies, San Francisco, CA
- 19 - 21 June 2019, Annual conference of the British Society for Gene and Cell Therapy (BSGCT), Sheffield, UK
- 25 - 26 June 2019, 4th PDA Europe Annual Meeting - gene therapy regulatory issues, Amsterdam, The Netherlands
- 20 - 23 July 2019, The 25th Annual Meeting of the Japan Society of Gene Therapy (JSGT), Tokyo, Japan
- 23 - 25 July 2019, 2nd Neuropsychiatric Drug Development NDD meeting - Neural Gene Therapy, Boston, MA
- 12 - 16 August 2019, The Bioprocessing Summit 2019, Boston, MA
- 13 - 15 August 2019, Blood-Brain Barrier Delivery Summit (B3DD) - Gene delivery, Boston, MA
- 9 - 12 September 2019, Cell & Gene Therapy Manufacturing and Commercialization, Boston, MA
- 10 - 13 September 2019, CAR-TCR Summit Boston 2019, Boston, MA
- 9 October 2019, The Cell & Gene Therapy Strategy Meeting Europe, Zurich, Switzerland
- 15 – 16 October 2019, 9th Annual Orphan Drugs and Rare Diseases conference, London, UK
- 15 – 17 October 2019, Gene Therapy for Rare Disorders Europe 2019, London, UK
- 21 – 22 October 2019, NORD's 2019 Rare Diseases & Orphan Products Breakthrough Summit, Washington, D.C.
- 21 – 25 October 2019, XXVII Congress of the European Society of Gene and Cell Therapy (ESGCT), Barcelona, Spain
- 24 – 25 October 2019, 5th Annual Cell & Gene Therapy Congress, London, UK
- 29 – 31 October 2019, Cell and Gene Therapy Manufacturing Forum event, London, UK
- 19 – 20 November 2019, 3rd Annual Ophthalmic Drugs Conference - gene therapy for eye diseases, London, UK
- 12 - 15 May 2020, American Society of Gene and Cell Therapy (ASGCT) 23th Annual Meeting, Boston, MA
- 31 March - 2 April 2020, 15th Annual World Advanced Therapies Congress, London, UK
- 20 – 23 October 2019, XXVIII Congress of the European Society of Gene and Cell Therapy (ESGCT), Edinburgh UK


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 and Zika Virus Net.
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