Inborn Errors of Metabolism Drug Development Summit 2020

Events
Inborn Errors - Pharmaphorum Banner

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

As 2020 continues, the number of therapies for inherited metabolic diseases moving into clinical development sits at an all-time high. Despite such growth, there remains a set of complex hurdles associated with drug development for rare metabolic diseases.

Inborn Errors of Metabolism Drug Development 2020 is dedicated to addressing these challenges to ensure that drug developers can achieve their full commercial potential.

Access the full event guide here to see how this meeting will seek to address your key challenges.

Join over 80 attendees and 24 speaking experts and learn how to:

  • Optimize your clinical trial design pathways with insights from Spark Therapeutics, Lysogene & Comet Therapeutics
  • More effectively monitor disease progression and therapeutic outcomes by designing superior clinical endpoints with insights from MinoryxSigilon Therapeutics
  • Maximize your patient engagement, access and retention for inherited metabolic disease clinical trials with insight from The NPKU Alliance, BioMarin & Sangamo Therapeutics
  • Address approval, investment and reimbursement challenges of drug development in order to improve early stage commercial decision making with insights from Sanofi & Homology Medicines

Click here to view the full agenda and speaker line up to see how you can accelerate your development of better effective treatments for inborn errors of metabolism.

This meeting will span across 3 full days including, 3 discussion-based workshops2 full summit days with in-depth case studies and learnings brought to you by the decision-making experts of the industry.

Hanson Wade

Hanson Wade

27 January, 2020