Welcome to Gene Therapy for Metabolic Disorders

Gene therapy and metabolic disease drug development each bring their own challenges. Gene Therapy for Metabolic Disorders will delve into the unique hurdles that companies are facing at the interface between the two, including: 

  • Determining first-in-human dosing from a preclinical program  
  • Defining and measuring relevant endpoints that have meaning to patients and regulators 
  • Re-creating specific target organ environments in preclinical models for more effective translation to humans 
  • Innovating clinical trial designs for rare pediatric populations 
  • Investigating liver-targeted therapies to achieve systemic clinical impact 
  • Targeting beyond the liver to transform treatment of neurometabolic diseases

Attendees of Other Gene Therapy Event Series Digital Meetings Have Said:

"It was very informative and
worked exceptionally well as a remote meeting." 

Novartis Gene Therapies 


“The event was a terrific overview of the gene therapy field, and was a great opportunity to see how other organizations view and address many relevant challenges.” 

Sarepta Therapeutics