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8.30am - 12.30pm

about the workshop

Investigation of the therapeutic potential of antisense oligonucleotides has in the main been confined to knocking down protein expression via RNAi or RNAse H.  In this workshop we discuss the role of AONs to treat disease by splice modification.  Prosensa has a portfolio of such products in the neuromuscular orphan disease area, with the lead compound in phase III trials together with our corporate partners GSK. Principles of drug design and development will be presented.  The personalized approach to therapy in an orphan disease raises a number of developmental and regulatory issues which will be discussed.

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about the leaders

 

 

Giles Campion, Chief Medical Officer and Senior Vice-President R&D is responsible for the company’s discovery and development strategy and activities. Board certified in rheumatology, he has more than 20 years of experience in the pharmaceutical and biotech industry and is an expert in translational medicine. He has held posts of increasing seniority in both large pharmaceutical and biotech companies working in Europe and the US, covering many different therapeutic areas.
During his many years in the industry, Dr Campion has been involved in the development of over 40 therapeutics and diagnostic agents and has led major filings and approvals in the US and Europe, including in the rare disease space.

Judith van Deutekom, Vice-President Drug Discovery is a molecular biologist and has an extensive 17-year track record in the genetic research of muscular dystrophies. She has been dedicated to the development of a genetic therapy for Duchenne Muscular Dystrophy (DMD) since her postdoctoral studies at the University of Leiden (The Netherlands) and the University of Pittsburgh Medical Center (Pennsylvania, USA). She was awarded for her research on antisense oligoribonucleotides as small molecule drugs for DMD by the Princess Beatrix fund in 2001 and by the LUMC (C.J. Kok Award) in 2003.

 

 

Sjef de Kimpe, Vice-President Early Drug Development is responsible for pre-clinical and early drug development. Prior to joining Prosensa, Sjef was the project manager for a multidisciplinary drug development team bringing a lead compound from academic research towards phase IIa in an orphan drug indication. He was also a life science consultant on IP, licensing and technology assessment. Sjef has over 18 years experience in pharmaceutical R&D. He was assistant professor of pharmacology and worked as a senior scientist with Nobel laureate Sir John Vane. Sjef studied pharmacy, holds a Ph.D in pharmacology and obtained an MBA at the NIMBAS University.
 

about the organisation

Prosensa is a biopharmaceutical company focused on the discovery, development and commercialization of RNA modulating therapeutics. The company targets genetic disorders with a large unmet medical need, with a primary focus on neuromuscular disorders.

 

Workshop programme

8:30 Registration & Coffee

9:00 Welcome & Introductions

Giles Campion

Giles Campion, Chief Medical Officer and Senior Vice-President R&D, Prosensa

9:10 Overview/ Splicing modification and oligo design

Judith van Deutekom

Judith van Deutekom, Vice-President Drug Discovery, Prosensa

  • Potential of AON to treat a variety of diseases through splicing modification
  • The principle of oligonucleotide design
  • Delivery, delivery, delivery
  • Animal models and preclinical proof of concept
  • 9:45 Preclinical safety and ADME of oligonucleotides

    Jessica Sipkens

    Jessica Sipkens, Scientific Manager Bioanalysis & DMPK , Prosensa

  • First and later generations of oligonucleotides and their class profile
  • Specific aspects of safety and relevance to man
  • Distribution, Metabolism and pharmacokinetics
  • Pharmacokinetic - pharmacodynamic modeling for clinical trials
  • 10:30 Morning Coffee

    11:00 A clinical case study – Duchenne muscular dystrophy

    Giles Campion

    Giles Campion, Chief Medical Officer and Senior Vice-President R&D, Prosensa

  • Duchenne Muscular Dystrophy – a devastating disease of dystrophin deficiency
  • Clinical proof of concept
  • Development in the orphan drug space
  • Regulatory consequences of a personalized approach
  • 12:00 Discussion session

    12:30 Close of Workshop

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    Copthorne Tara Hotel

    Scarsdale Place
    Kensington
    London W8 5SR
    United Kingdom

    Copthorne Tara Hotel

    The Copthorne Tara Hotel London Kensington is an elegant contemporary four-star hotel in prestigious Kensington, located just a two minutes walk from High Street Kensington underground station, making exploring easy. The hotel offers well-appointed and comfortable guest rooms combining Standard, Superior and Club accommodation. Club rooms offer iconic views over the city and include Club Lounge access for complimentary breakfast and refreshments. Guests can sample the authentic Singaporean, Malaysian and Chinese cuisine at Bugis Street, traditional pub fare at the Brasserie Restaurant & Bar or relax with a delicious drink at West8 Cocktail Lounge & Bar.

    The Copthorne Tara Hotel boasts 745 square meters of flexible meeting space, consisting of the Shannon Suite and the Liffey Suite, ideal for hosting conferences, weddings and social events. Facilities include access to the business centre 24 hours a day, fully equipped fitness room, gift shop, theatre desk and Bureau de Change. With ample onsite parking outside the London congestion charge zone and excellent transport links via Heathrow Airport, the hotel is the perfect location for business or leisure stays. The hotel is within close proximity to the shops of High Street Kensington, Knightsbridge and Westfield London, Olympia Conference Centre, Royal Albert Hall, Kensington Palace and Hyde Park.

     

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    WHAT IS CPD?

    CPD stands for Continuing Professional Development’. It is essentially a philosophy, which maintains that in order to be effective, learning should be organised and structured. The most common definition is:

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