Confidently Translate, Develop & Commercialize Gene, mRNA, Replacement Therapies, Small Molecule & Substrate Reduction Therapies to More Efficaciously Treat Inherited Metabolic Diseases

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.

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Access the full 2-day agenda, workshops, speaker line-up, partner information, pricing and discounts

World-Class Speakers

Adith Venkiteshwaran

Global New Product Planning, Rare Diseases

Sanofi

David Pauza

Chief Scientific Officer

American Gene Technologies

Katherine Cygnar

Senior Staff Scientist

Regeneron

Mathias Schmidt

CEO

ArmaGen

Paul Humphrey

Director Patient Advocacy

BioMarin

3 Key Reasons to Attend

  • Engage directly with the leading experts from pioneering pharma and biotech companies to gain first-hand experience on how to accelerate your drug development process
  • Understand how to navigate the fundamental clinical, regulatory, and patient access hurdles currently prohibiting accelerated drug development and commercialization for novel rare metabolic disease therapeutics
  • Immerse yourself in 3 days of in-depth talks and interactive sessions focusing specifically on rare metabolic disease drug development

Hear What Attendees Say

“The quality of attendees are what make a great conference and the people attending this meeting were absolutely first-rate.

Adith Venkiteshwaran, Global New Product Planning, Rare Diseases, Sanofi

 

“This conference is a good opportunity for industry leaders to be able to come together and learn how different companies will tackle challenges from different perspectives, and it allows us to learn from each other”

Vivian Choi, Associate Director, Head of Gene Therapy Research, Shire Pharmaceuticals

 

"I really enjoyed the broad spectrum of topics that the conference covered and the networking opportunities"

Samantha Galuska, Senior Research Associate, Sanofi

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