Gene Therapy for Rare Disorders 2021 | 100% Digital

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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 investment at an all-time high, and several indications in late-stage trials gearing up for approval, 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 90+ industry-leading speakers, this digital 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 500+ of your colleagues online to accelerate the progress of the next generation of gene therapies.

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


Time: 07:00 to 18:00

FULL ACCESS PASS: 2 Day Conference + Workshop Day AND Discussion Day - Drug Developer Pricing: USD 4097.00,
2 Day Conference + Workshop Day OR Discussion Day - Drug Developer Pricing: USD 3298.00,
2 Day Conference - Drug Developer Pricing: USD 2499.00,
FULL ACCESS PASS: 2 Day Conference + Workshop Day AND Discussion Day - Standard Pricing: USD 4797.00,
2 Day Conference + Workshop Day OR Discussion Day - Standard Pricing: USD 3998.00,
2 Day Conference - Standard Pricing: USD 3199.00

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|-100%-Digital

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Dearing, KS 67340


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