THIS SITE IS INTENDED FOR HEALTHCARE PROFESSIONALS ONLY.

Sponsorship statement: This is a non-promotional meeting funded and organised by Sarepta. These presentations and discussions are for educational purposes only and solely intended to provide scientific information on certain diseases. Nothing in these presentation and discussions should be construed as seeking to promote any of Sarepta’s therapies. Nothing in these presentations should be disclosed externally without Sarepta’s prior agreement.

Practical experiences from running an adeno-associated virus gene therapy trials: The Principal Investigator’s perspective

XX September 2020 | 18:00 GMT/19:00 CET

Live Webinar

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Pan-European Series

Managing gene therapy trials: Sharing best practice

Practical experiences from running an adeno-associated virus gene therapy trials: The Principal Investigator’s perspective

The importance of gene therapy in major therapy areas is increasing year on year, and consequently gene therapy trials are being instigated. Yet relatively few centres across Europe have experience of running clinical trials in this complex area.

This series of webcasts will provide a forum for sharing best practice on the core aspects of running AAV gene therapy trials. The series begins with a webcast on the background to gene therapy trials, the differences between these and traditional clinical trials, and the key advice you need to establish a new trial site. The webcast will include presentations by experts from across Europe, as well as panel discussions and the opportunity for you to pose your questions to the experts in live Q&A sessions.

Practical experiences from running an adenoassociated virus gene therapy trials: The Principal Investigator’s perspective

XX September 2020 | 18:00 GMT/19:00 CET

Speakers

Professor Francesco Muntoni

Professor of Paediatric Neurology, Director of the Dubowitz Neuromuscular Centre, at the UCL Institute of Child Health and Great Ormond Street Hospital for Children, London, UK

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Professor Eugenio Mercuri

Professor of Paediatric Neurology at the Catholic University, Rome, Italy

Lorem ipsum dolor sit amet, consectetur adipiscing elit. Ut elit tellus, luctus nec ullamcorper mattis, pulvinar dapibus leo.

Professor Laurent Servais

Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre, Invited Professor of Child Neurology at Liège University.

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Professor Nicolas Deconick

Chef de clinique neurologie pédiatrique Hôpital Universitaire Des Enfants Reine Fabiola (HUDERF), Brussel, Belgium

FULL BIO +

Professor Wolfgang Müller-Felber

Head of the centre of pediatric neuromuscular diseases at the v. Hauner children hospital, University of Munich, Munich, Bavaria, Germany

FULL BIO +

Speakers

Professor Francesco Muntoni

Professor of Paediatric Neurology, Director of the Dubowitz Neuromuscular Centre, at the UCL Institute of Child Health and Great Ormond Street Hospital for Children, London, UK

Lorem ipsum dolor sit amet, consectetur adipiscing elit. Ut elit tellus, luctus nec ullamcorper mattis, pulvinar dapibus leo.

Professor Eugenio Mercuri

Professor of Paediatric Neurology at the Catholic University, Rome, Italy

Lorem ipsum dolor sit amet, consectetur adipiscing elit. Ut elit tellus, luctus nec ullamcorper mattis, pulvinar dapibus leo.

Professor Laurent Servais

Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre, Invited Professor of Child Neurology at Liège University.

Lorem ipsum dolor sit amet, consectetur adipiscing elit. Ut elit tellus, luctus nec ullamcorper mattis, pulvinar dapibus leo.

Professor Francesco Muntoni

Professor of Paediatric Neurology, Director of the Dubowitz Neuromuscular Centre, at the UCL Institute of Child Health and Great Ormond Street Hospital for Children, London, UK

Lorem ipsum dolor sit amet, consectetur adipiscing elit. Ut elit tellus, luctus nec ullamcorper mattis, pulvinar dapibus leo.

Professor Eugenio Mercuri

Professor of Paediatric Neurology at the Catholic University, Rome, Italy

Lorem ipsum dolor sit amet, consectetur adipiscing elit. Ut elit tellus, luctus nec ullamcorper mattis, pulvinar dapibus leo.

Agenda

25 minutes

Gene therapy trials – challenges and opportunities

  • Welcome, intro to the meeting and agenda, instructions for delegates

  • A brief history of gene therapy trials in Europe

  • Gene therapy trials vs traditional trials – what are the differences?

  • Reflections on successful gene therapy trials

  • Practical guidance on running multiple gene therapy trials at a single site

Speaker(s):
Prof Francesco Muntoni
Prof Eugenio Mercuri

15 minutes

Establishing a new trial site

  • Transferrable knowledge: how learnings from one site can be carried across to another
  • Priorities when establishing a new site

Speaker(s):
Prof Laurent Servais

20 minutes

Panel discussion

Personal experiences of trial management – what worked and what didn’t?

  • What similarities and differences are there between the approaches taken in the different countries?
  • ‘Top tips’ – what piece of advice would the faculty give to PIs initiating a new gene therapy trial?
  • Trial troubleshooting – what barriers/challenges are common and how could these be overcome?

Speaker(s):

Prof Francesco Muntoni / Prof Eugenio Mercuri (chairs)

Prof Laurent Servais / Prof Nicolas Deconick /
Prof Wolfgang Müller-Felber (faculty)

30 minutes

Live Q&A

Chairs:
Prof Francesco Muntoni
Prof Eugenio Mercuri

Book your place at the webinar

Practical experiences from running an adeno-associated virus gene therapy trials: The Principal Investigator’s perspective

XX September 2020 | 18:00 GMT/19:00 CET

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Sponsorship statement:
This is a non-promotional meeting funded and organised by Sarepta. These presentations and discussions are for educational purposes only and solely intended to provide scientific information on certain diseases. Nothing in these presentation and discussions should be construed as seeking to promote any of Sarepta’s therapies. Nothing in these presentations should be disclosed externally without Sarepta’s prior agreement.

Job code: XXX | Date of preparation: August 2020

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Practical experiences from running an adeno-associated virus gene therapy trials: The Principal Investigator’s perspective

XX September 2020

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