About Event

Hanson wade have taken the decision to cancel this meeting. Please do accept our apologies for any inconvenience or disappointment this will cause. Please register your interest if you would like updates on the meeting or topic.

WELCOME TO THE 2ND ANNUAL INBORN ERRORS OF METABOLISM
DRUG DEVELOPMENT SUMMIT

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.

  • Working Alongside Clinicians and Regulators to Identify and Diagnose Patients with Inborn Errors of Metabolism
  • Clinical Endpoints & Biomarkers: Monitoring Disease and Therapeutic Outcomes
  • Clinical Trial Design, Patient Stratification and Ethics
  • Optimizing Enzyme Replacement and mRNA Therapy
  • Collaboration within the field as well as with Large Pharma Companies and Patient Advocacy Groups to Advance Research and Reduce Unmet Clinical Needs Around the World
  • Cell & Gene Therapy Methods and Challenges
  • Preclinical Development of Novel IEM Therapeutics
  • Tissue, Organ and Systemic Therapeutic Delivery