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Bluebird bio, ReGenX, uniQure to Speak at Orphan Drugs Conference, Feb. 20-21, 2014, SD, CA

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2013-12-10 00:22:39 - The Orphan Drugs Research & Commercialization and the Cell & Gene Therapy Conferences, to be held February 20-21, 2014 in San Diego, CA will feature a special joint session on “Cell and Gene Therapy for Rare Diseases: From Research to Regulatory.”

Over the past years, gene and cell therapy have emerged as promising treatments for those with rare diseases. In November 2012, Netherlands-based uniQure became the first company to have its gene therapy treatment approved, when Glybera was given the green light by the European Medicines Agency.

The session will open with plenary addresses from Stephen Groft, Director of the Office of Rare Diseases at the NIH, and Harald Petry, Chief Scientific Officer at uniQure. The session will then include presentations from the following speakers:

• Mitch Finer, Chief Scientific Officer, bluebird bio
• Philip Gregory, Chief Scientific Officer, Sangamo Biosciences
• Brian Kaspar, Principal Investigator, Center for Gene Therapy, Nationwide Children’s Hospital
• Karen Kozarsky, Vice President of Research & Development, ReGenX Biosciences
• Trent

Spencer, Assistant Professor of Pediatrics, Emory University School of Medicine

The speakers will cover the various aspects of cell and gene therapy, from using adeno-associated viral (AAV) gene therapy as a platform to treat rare diseases, conducting translational studies using gene therapy, overcoming regulatory barriers, and much more.

The Orphan Drugs Research & Commercialization Conference is a forum for various stakeholders in the community to gather and share complementary perspectives on the challenges in the field and paths forward. The meeting includes presentations on the latest scientific therapeutics, panel discussions on patient registries and advocacy, networking sessions, and much more. The conference will run in parallel with the Cell & Gene Therapy Conference as part of the larger Future Biotech Summit.

For more information, please visit

Contact Information:

635 W. Foothill Blvd. Monrovia, CA 91016

Contact Person:
Kristen Starkey
Conference Production
Phone: (626) 256-6405
email: email


Rania hafez
Phone: 626-256-6405

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