18 December 2019

Pharma Integrates is a unique event which brings together key industry leaders from the Pharmaceutical pipeline to share insights and create debate on topics which impact the future of patient outcomes. This year the conference took place on the 18th and 19th November 2019 in London and welcomed 90 speakers, 34 sponsors and over 450 delegates.


tranScrip’s Head of Rare Diseases & Advanced Therapeutics, Gilbert Wagener, was in attendance and made an excellent contribution to the panel of the session entitled ‘The changing winds in rare diseases’. How to navigate the largely unchartered territory of the emerging field of rare diseases was the topic up for discussion.

Gilbert commented, 'Pharma Integrates offered an excellent forum to discuss and to understand the particular aspects of orphan drug development with colleagues and affected parents and on how to improve the development of orphan drugs. 80% of orphan diseases are monogenetic and therefore the role of gene therapy in orphan drug development is crucial. True innovation at affordable costs will be the challenge to improve patients´ access to effective medicines.'

Gilbert is an expert in immunological based drug development, cardiovascular and rare/orphan disease and has over 20 years’ experience in global drug development in a wide range of areas.

Out of all the sessions at the conference, ‘The changing winds in rare disease’ received the highest number of impressions as showed by a post event social media report. Recordings of the session are available here.

Other panellists in the session include:
Ron Jortner (Facilitator) – Managing Director and Trustee at Masthead Biosciences & Cambridge Rare Disease Network
Owen MarksRare Disease Lead at Pfizer
Paul Stott Chief Executive Office of Seda Pharmaceutical Development Services
Daniel LewiFounder of Cure & Action for Tay-Sachs (CATS) Foundation
Shelley WatchamAssociate Medical Director at Sobi

More information can be found at:
Pharma Integrates 2019 press release
Life Science Integrates
Pharma Integrates 2019 Recordings
– Track 3
Gilbert Wagener’s profile