Advancing Gene Therapy  

Objectives:

• Control the expression of genetic causes of CMT through gene editing, silencing, epigenetics, splice modification, and other approaches, including both gene therapy and small molecule approaches
• Understand and control modifier genes that impact disease severity and/or progression in CMT

Promoting Nerve Health                    

Objectives:

• Understand and pharmacologically control cellular and molecular pathways that affect axon health, including survival, regeneration, and target connectivity
• Understand and pharmacologically control cellular and molecular pathways that affect peripheral myelination, including Schwann cell survival and function

Overcoming the Delivery Challenge 

Objectives:

• Understand and manipulate the peripheral nerve barriers (epineurium, perineurium, endoneurium, and blood-nerve barrier)
• Develop novel delivery mechanisms
• Refine existing delivery mechanisms

De-risking Clinical Trials (Secondary Priority)                    

Objectives:

• Increase the rigor of CMT research with an eye toward enhancing its drug development value
• Expand data and sample sharing in CMT research, including access for industry researchers
• Define the health economics of CMT, including global incidence/prevalence, economic burden, and effect of therapeutics
• Further the development of existing or novel outcome measures that respond rapidly to changes in disease progression and treatment
• Further the development of existing or novel biomarkers for disease progression, target engagement, and treatment response