Gene Therapy for Rare Disorders 2020

Gene therapies are redefining the treatment of rare diseases. However, a set of complex and unique challenges must be overcome for these therapeutics to achieve their commercial potential.

The 4th Annual Gene Therapy for Rare Disorders will focus exclusively on overcoming the late-stage commercial challenges drug developers face when delivering gene therapies to market.

With approvals in a number of indications over the last year, and more rapidly approaching on the horizon, the meeting will leverage the experience from first to market pioneers, as well as unveiling the strategies
drug developers are employing to improve efficacy, safety and commercial viability.

Incorporating insights from 80+ industry-leading speakers, this conference will delve into the key regulatory, reimbursement, clinical and manufacturing hurdles that need to be overcome to realize the commercial potential of gene therapies.

Join 600+ of your colleagues to accelerate the progress of the next generation of gene therapies.

This is your comprehensive guide to define your commercial path forward in 2020.

Early booking and group discounts apply. Please visit the website for more information.

URLs:
Tickets: https://go.evvnt.com/547519-2?pid=5248
Brochure: https://go.evvnt.com/547519-3?pid=5248

Speakers: Mohammed Asmal, VP Head of Clinical Research And Development, bluebird bio, Pamela Bradt, CSO, ICER, Daniel Chung, Medical Affairs And Clinical Ophthalmic Lead, Spark Therapeutics, Katherine Dallow, VP, Clinical Programs And Strategy, Blue Cross Blue Shield of Massachusetts, Olivier Danos, CSO, REGENXBIO, Xiaotong Fu, Senior Engineer, Biogen, Mark Galbraith, Head of Quality Control And Analytical Sciences, Spark Therapeutics, Kathleen Hehir, Head of Gene Therapy Clinical Manufacturing, Sanofi, Kei Kishimoto, CSO, Selecta Biosciences, Keith Life, Senior Director, Gene Therapy Manufacturing And Viral Vector Production, BioMarin, Julie Lin, Global Project Head of Early Clinical And Business Development, Sanofi, Janet Lynch Lambert, CEO, Alliance for Regenerative Medicine (ARM), Jason Mallory, Clinical Development Leader, Spark Therapeutics, Luis Maranga, CTOO, Voyager Therapeutics, Peter Marks, Director, CBER, FDA, Chris Mason, CSO, AvroBio, Emily McGinnis, VP, Patient Advocacy And Professional Relations, AveXis, Federico Mingozzi, CSO, Spark Therapeutics, Palani Palaniappan, SVP And Head of Global Technical Operations, Sarepta Therapeutics, Robert Pietrusko, Senior Vice President, Regulatory Affairs And Quality Assurance, Voyager Therapeutics, Philip Reilly, Venture Partner, Third Rock Ventures, Michael Sherman, CMO And SVP, Harvard Pilgrim Health Care Institute, Mark Trusheim, Strategic Director, NEWDIGS And Visiting Scientist, MIT, Gabor Veres, VP, Head of Gene Therapy, BioMarin, Sam Wadsworth, CSO, Ultragenyx, Yogesh Waghmare, Associate Director And Head of Downstream Vector Process Development And CMC Lead, bluebird bio, James Warren, VP, Pharmaceutical Development, Ultragenyx, Jill Weimer, SVP Discovery Research and Gene Therapy Science, Amicus Therapeutics, Fraser Wright, Professor, Pediatrics, Stanford University School of Medicine And CTO, Axovant, Alex Xu, Chief Scientist, CFDA

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