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 presentations and discussions should be construed as seeking to promote any of Sarepta’s therapies. Nothing in these presentations should be disclosed externally without the prior agreement of Sarepta.

12 January 2021 | 18:00 GMT/19:00 CET

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Pan-European Faculty

Practical experiences from running gene therapy trials with
Adeno-Associated Virus (AAV) vectors: The Primary Investigator's perspective

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

This online event is a new initiative from Sarepta, and will provide a forum for sharing best practice on the core aspects of running AAV gene therapy trials – including the background to these trials, 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 a live Q&A session.

Practical experiences from running gene therapy trials with Adeno-Associated Virus (AAV) vectors: The Primary Investigator's perspective

12 January 2021 | 18:00 GMT/19:00 CET

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Agenda

Session 1

Gene therapy trials – challenges and opportunities

15 minutes

  • Welcome, introduction to the meeting and agenda, instructions for delegates
  • Principles and possibilities of gene therapy
  • Gene therapy trials vs. traditional trials – what are the differences?

  • Practical guidance on running gene therapy trials

Speaker:
Prof Eugenio Mercuri

Session 2

Principles and applications of gene therapy

15 minutes

  • A brief history of gene therapy trials in Europe
  • Reflections on successful gene therapy trials

Speaker:
Prof Francesco Muntoni

Session 3

Establishing a new trial site

15 minutes

  • Transferrable knowledge: how learnings from one site can be carried across to another
  • Priorities when establishing a new site
  • Establishing multiple gene therapy trials at a single site

Speaker:
Prof Laurent Servais

Session 4

Panel discussion

15 minutes

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?
Speakers: Prof Francesco Muntoni and Prof Eugenio Mercuri (chairs) 
Prof Laurent Servais, Prof Nicolas Deconinck and Prof Wolfgang Müller-Felber (faculty)

Session 5

Live Q&A

30 minutes

Chairs:
Prof Francesco Muntoni and Prof Eugenio Mercuri

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12 January 2021 | 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 presentations and discussions should be construed as seeking to promote any of Sarepta’s therapies. Nothing in these presentations should be disclosed externally without the prior agreement of Sarepta.

Job code: MED-GBR-GT-0002 | Date of preparation: December 2020

© 2020 SAREPTA THERAPEUTICS. CONFIDENTIAL AND NOT FOR DISTRIBUTION.
COPYING OF THIS MATERIAL BY ANY MEANS WITHOUT THE PRIOR WRITTEN CONSENT OF SAREPTA IS PROHIBITED.

SAREPTA, SAREPTA THERAPEUTICS and the SAREPTA Helix Logo are trademarks of Sarepta Therapeutics, Inc. registered in the U.S. Patent and Trademark Office and may be registered in various other jurisdictions.

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12 January 2021 | 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 presentations and discussions should be construed as seeking to promote any of Sarepta’s therapies. Nothing in these presentations should be disclosed externally without the prior agreement of Sarepta.

Job code: MED-GBR-GT-0002 | Date of preparation: December 2020

SAREPTA, SAREPTA THERAPEUTICS and the SAREPTA Helix Logo are trademarks of Sarepta Therapeutics, Inc. registered in the U.S. Patent and Trademark Office and may be registered in various other jurisdictions.