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Abeona to Explore Current Trends Within Orphan Drugs Development at Orphan Drugs & Rare Diseases 2016

SMi Reports (, London, UK): Abeona President & CEO to review opportunities and challenges in orphan drug development at Orphan Drugs & Rare Diseases 2016.

London, United Kingdom, August 24, 2016 --( Abeona has joined the expert speaker line-up for the 5th annual conference on Orphan Drugs and Rare Diseases which takes place on 19 & 20 October 2016 in Central London.

Tim Miller PhD, President and CEO, will be will chairing the two-day event.

Dr Miller will be speaking at the conference on the “Development of orphan drugs to prevent, diagnose and treat rare diseases.” His presentation will review the challenges of orphan drugs development and creating workable solutions to overcome them. The talk will also explore ways to establish early access to orphan drugs and analyse what regulatory bodies can do to support potential investors.

Dr Miller will also be leading a panel discussion on how partnerships can improve the orphan drugs landscape. Other panellists include Nigel Nicholls, Country Director at BioMarin; Robert Karl, Partner at RBV Capital; Michelle Berg, Vice President Patient Advocacy at Abeona; and Daniel O’Connor, Medical Assessor at MHRA.

In the run-up to the event, SMi Group spoke to Dr Miller about the recent developments impacting the orphan drug landscape. “AAV gene therapy is transforming how we look at and treat patients with rare diseases – we’re now able to target genes for replacement or editing using these very specific delivery vehicles. Gene therapy is all about delivery, and the impact we’re seeing in clinical trial success will set the stage for personalizing genomic medicine.”

When asked about the biggest challenge for the industry, he responded, "The biggest challenge in rare and orphan disease is manufacturing. Once proof of concept has been demonstrated pre-clinically, there can be significant scale-up challenges when moving from 1-10 patients to 200-500 patients."

To read the full interview with Dr Miller, visit the Download Centre on the event website at

Other speakers include Sheela Upadhyaya, Associate Director at NICE; Tony Hall, Therapeutic Area Head for Orphan Diseases at Mereo BioPharma; Cecile de Coster, Associate Director of Regulatory Affairs at Alexion; and many more. A complete list of speakers and their topics is available to view on the event website.

For those interested in attending Orphan Drugs 2016, registration is live online at

For those interested to speak or to exhibit at the event, please contact Alia Malick on +44(0)207 827 6168 or For delegate enquiries, please contact Matt Apps on +44(0)207 827 6093 or

For media enquiries please contact Honey de Gracia on +44(0)207 827 6102 or

5th Orphan Drugs Conference and Exhibition
19-20 October 2016, London UK

About SMi Group:

Established since 1993, the SMi Group is a global event-production company that specializes in Business-to-Business Conferences, Workshops, Masterclasses and online Communities. We create and deliver events in the Defence, Security, Energy, Utilities, Finance and Pharmaceutical industries. We pride ourselves on having access to the world’s most forward thinking opinion leaders and visionaries, allowing us to bring our communities together to Learn, Engage, Share and Network. More information can be found at
Contact Information
SMi Group
Honey de Gracia
+44 (0)20 7827 6102

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