Inborn Errors of Metabolism Drug Development Summit 2020

Contact Name:
Fergus Cunningham
+1 617 455 4188
Filed in:
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Join over 80 inherited metabolic disease drug developers and KOLs to explore how the key challenges prohibiting accelerated drug development are being addressed. The inaugural Inborn Errors of Metabolism Drug Development Summit will provide a discursive platform to examine where pioneering gene therapy modalities intersect with current therapeutic options and explore how diseases can be better targeted in order to address the huge unmet medical need for rare metabolic diseases.

With scarce treatment options leaving inborn errors of metabolism a devastating, unfilled and unmet medical need, the opportunity for biopharma to do better and aggressively tap into a lucrative and life changing market of gene modalities is growing. For the first time, the Inborn Errors of Metabolism Drug Development summit will look to examine how the experience within existing pharmacology for rare metabolic diseases can be leveraged with the drug development strategies of those pioneering gene modalities to improve efficacy, safety and commercial viability.

Incorporating insights from industry-leading speakers, this conference will delve into the key patient access, regulatory, clinical and commercial hurdles that need to be overcome in order to address the unmet medical need of rare metabolic diseases and fulfil their full commercial potential.

This is your comprehensive guide to define your drug development path for 2020. Don’t miss out on your chance to network with industry leaders and be a part of the only enriched industry forum focused on accelerating the development of better effective treatments for inborn errors of metabolism.



Conference + 3 Workshops (Drug Developer): USD 4396.00
Conference + 2 Workshops (Drug Developer): USD 3897.00
Conference + 1 Workshop (Drug Developer): USD 3398.00
Conference Only (Drug Developer): USD 2899.00
Workshops Only (Drug Developer): USD 599.00
Conference + 3 Workshops (Academic): USD 3079.00
Conference + 2 Workshops (Academic): USD 2729.00
Conference + 1 Workshop (Academic): USD 2379.00
Conference Only (Academic): USD 2029.00
Workshop Only (Academic): USD 419.00

Speakers: Paolo Martini, CSO, Moderna Therapeutics, Klaus Gregorius, Co-Founder and CSO, MipSalus, Chris Carpenter, CMO, Rubius Therapeutics, David Pauza, Chief Scientific Officer, American Gene Technologies, Katherine Cygnar, Senior Staff Scientist, Regeneron, Deya Corzo, CMO, Sigilon Therapeutics, Dustin Armstrong, CSO, Valerion Therapeutics, Sonia Poli, CSO, Minoryx, Mathias Schmidt, CEO, ArmaGen, Neil Smith, CMO, Censa Pharmaceuticals, Art Taveras, CSO, Comet Therapeutics, Lex Cowsert, Director of Research, National PKU Alliance, Julie Eisengart, Assistant Professor, Department of Pediatrics, University of Minnesota, Paul Humphrey, Director Patient Advocacy, BioMarin, Reena Kartha, Associate Director Translational Pharmacology, Center for Orphan Drug Research, University of Minnesota, Christeen Moburg, Director Patient Advocacy, Sangamo Therapeutics, Michael Blum, Vice President, Commercial Strategy, Homology Medicines, Adith Venkiteshwaran, Global New Product Planning, Rare Diseases, Sanofi

Time: 8:00 am to 9:00 pm

Street Address

Courtyard Boston Downtown, 275 Tremont Street
Boston, MA 02116


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