RegenXbio's Duchenne Gene Therapy Secures FDA Orphan Drug Tag

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The FDA has granted Orphan Drug Designation to RegenXbio Inc's RGNX RGX-202, potential one-time gene therapy for Duchenne muscular dystrophy (Duchenne). 

  • Duchenne is a genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin that helps keep muscle cells intact.
  • RGX-202 is designed to deliver optimized microdystrophin transgene with a unique C-terminal domain and a muscle-specific promoter to support targeted therapy for improved resistance to muscle damage associated with Duchenne. 
  • RGX-202 uses RegenXbio's proprietary NAV AAV8 vector. 
  • The Company expects to submit an Investigational New Drug (IND) application to the FDA for RGX-202 by the end of 2021. 
  • Related content: Benzinga's Full FDA Calendar.
  • Price Action: RGNX shares are up 0.66% at $33.73 during the market session on the last check Monday.
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