About Event


As the field of inherited metabolic disease looks to assess the viability of pioneering gene therapy modalities, drug development within existing pharmacology continues as second-generation enzyme replacement and small molecule therapies progress through the clinic. Regardless of the therapeutic modality, you aim to build, all drug developers are working to fulfill the huge unmet medical need that exists for inherited metabolic disorders. In order to accelerate drug development and address this unmet medical need, a set of unique and complex challenges associated with rare metabolic diseases must be overcome. If these challenges can be addressed drug developers can be confident that their therapeutic modalities will achieve their full commercial potential.

  • Explore novel and existing delivery pathways in order to more effectively bring your therapeutic to the site of action
  • Address patient access and recruitment in an increasingly competitive landscape to more effectively source the right patient for the right trial
  • Identify robust clinical endpoints and biomarkers to better understand disease progression and therapeutic outcome
  • Harness the power of natural history research and rethink your approach to clinical trial design to confidently produce meaningful clinical endpoints
  • Analyze current regulatory requirements to better navigate the regulatory landscape and more confidently progress your drug development pipeline
  • Address the approval, investment and reimbursement challenges of IEM drug development to accelerate your therapeutics from the clinic to commercialization
  • Highlight unmet medical needs for ultra-rare diseases to drive forward investment and accelerate drug development