U.S. markets closed
  • S&P 500

    4,397.94
    -84.79 (-1.89%)
     
  • Dow 30

    34,265.37
    -450.02 (-1.30%)
     
  • Nasdaq

    13,768.92
    -385.10 (-2.72%)
     
  • Russell 2000

    1,987.92
    -36.12 (-1.78%)
     
  • Crude Oil

    84.83
    -0.72 (-0.84%)
     
  • Gold

    1,836.10
    -6.50 (-0.35%)
     
  • Silver

    24.34
    -0.37 (-1.50%)
     
  • EUR/USD

    1.1348
    +0.0031 (+0.27%)
     
  • 10-Yr Bond

    1.7470
    -0.0860 (-4.69%)
     
  • GBP/USD

    1.3555
    -0.0045 (-0.33%)
     
  • USD/JPY

    113.6500
    -0.4500 (-0.39%)
     
  • BTC-USD

    35,743.38
    +242.66 (+0.68%)
     
  • CMC Crypto 200

    870.86
    +628.18 (+258.85%)
     
  • FTSE 100

    7,494.13
    -90.88 (-1.20%)
     
  • Nikkei 225

    27,522.26
    -250.67 (-0.90%)
     

RegenXbio's Duchenne Gene Therapy Secures FDA Orphan Drug Tag

  • Oops!
    Something went wrong.
    Please try again later.
·1 min read
In this article:
  • Oops!
    Something went wrong.
    Please try again later.
  • RGNX

The FDA has granted Orphan Drug Designation to RegenXbio Inc's (NASDAQ: 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.

See more from Benzinga

© 2021 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.