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

In the absence of physical meetings, the Inborn Errors of Metabolism team understands that there is still huge value to the inherited metabolic disease community in providing an alternative platform for knowledge sharing and scientific corroboration. As such, the Inborn Errors of Metabolism Drug Development summit has gone digital!

Join our community of inherited metabolic disease drug developers and KOLs to explore how the key challenges prohibiting accelerated drug development are being addressed. This 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 fulfill 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

What Does the New Digital Format Mean for You?

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Gain insights from 17 industry experts from the likes of ArmaGen, Moderna Therapeutics, Minoryx, Comet Therapeutics, and more, as they share their knowledge and expertise.

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Get involved in over 14 hours of content across three days as we delve into the industry's most pressing challenges.

  • Two discussion-led pre-conference workshops
  • 16 individual case studies
  • Multiple live Q&A sessions
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Networking Opportunities

Take part in over 3 hours of virtual networking sessions and use our online networking platform to connect with those industry leaders.

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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