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Joint Session on Rare Diseases at Cell & Gene Therapy Conf. Feb 2014, San Diego

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2013-12-16 05:32:52 - Joint Session on Rare Diseases at Cell & Gene Therapy Conf. Feb 2014, San Diego

GTC proud to present the Inaugural Cell and Gene Therapy Conference to be held on February 20-21, 2014 in San Diego, CA. There is a special joint session between this meeting and our co-located 2nd Orphan Drugs Research & Commercialization meeting, in which research to regulatory issues in cell and gene therapy for rare diseases will be discussed.

This session will include keynote presentations from Harald Petry, the Chief Scientific Officer of uniQure B.V. and Stephen Groft, Director of the Office of Rare Diseases Research at the National Institutes of Health.

Dr. Petry is currently the Chief Scientific Officer of uniQure B.V. uniQure’s Glybera® is the first gene therapy approved by the European Commission and by regulatory authorities in the Western

world. It is the first medication approved for patients with Lipoprotein Lipase Deficiency, a rare metabolic disorder.

Dr. Groft’s major focus is on stimulating research with rare diseases and developing information about rare diseases and conditions for health care providers and the public. To help identify research opportunities and establish research priorities, the Office has co-sponsored over 1200 rare diseases-related scientific conferences with the NIH research Institutes and Centers and the extramural research community, including patient advocacy groups. Dr. Groft’s presentation will focus on gene therapy for rare genetic diseases.

Other distinguished speakers in this special joint session include Mitchell Finer, Chief Scientific Offer at bluebird bio; Philip Gregory, the Chief Scientific Officer at Sangamo Biosciences; Karen Kozarsky, Vice President of Research and Development at ReGenX Biosciences, who will present on “AAV Gene Therapy as a Platform for the Treatment of Rare and Inherited Disease”; Brian Kaspar, Principal Investigator at the Center for Gene Therapy at Nationwide Children’s Hospital, who will discuss “Translational Studies in Neuromuscular Disease Using Gene Therapeutics”; and Trent Spencer, the Director of Gene Therapy at Emory University School of Medicine, who will discuss “Gene Therapy for Hemophilia A”.

Treatment of disease using cell and gene therapy is now a reality. With emerging technologies and new clinical trials being developed and launched at a growing rate, it is important to stay up-to-date with the evolving regulatory challenges and pre-clinical requirements for drug development and discovery. This meeting brings together experts from industry and academia to discuss the development and applications of cell and gene therapy as well as strategies and challenges for commercialization.

For most full speaker list click here

This conference is also part of the Future Biotech Summit 2014, which consists of this conference, and two other additional co-located conferences:
Cell & Gene Therapy
2nd Orphan Drugs Research & Commercialization
Ubiquitin Research & Drug Discovery

For more information, please visit

Contact Information:

635 W. Foothill Blvd. Monrovia, CA 91016

Contact Person:
Kristen Starkey

Phone: 6262566405
email: email


Rania hafez
Phone: 626-256-6405

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