U.S. Markets closed
  • S&P 500

    4,411.79
    +44.31 (+1.01%)
     
  • Dow 30

    35,061.55
    +238.20 (+0.68%)
     
  • Nasdaq

    14,836.99
    +152.39 (+1.04%)
     
  • Russell 2000

    2,209.65
    +10.17 (+0.46%)
     
  • Crude Oil

    72.17
    +0.26 (+0.36%)
     
  • Gold

    1,802.10
    -3.30 (-0.18%)
     
  • Silver

    25.24
    -0.14 (-0.56%)
     
  • EUR/USD

    1.1770
    -0.0003 (-0.0235%)
     
  • 10-Yr Bond

    1.2860
    +0.0210 (+1.66%)
     
  • Vix

    17.20
    -0.49 (-2.77%)
     
  • GBP/USD

    1.3754
    -0.0013 (-0.0977%)
     
  • USD/JPY

    110.5100
    +0.3950 (+0.3587%)
     
  • BTC-USD

    33,676.27
    +1,031.77 (+3.16%)
     
  • CMC Crypto 200

    786.33
    -7.40 (-0.93%)
     
  • FTSE 100

    7,027.58
    +59.28 (+0.85%)
     
  • Nikkei 225

    27,548.00
    +159.80 (+0.58%)
     

Voyager Therapeutics' Stock Jumps As FDA Lifts Clinical Hold For Huntington's Disease Gene Therapy

  • Oops!
    Something went wrong.
    Please try again later.
·1 min read
In this article:
  • Oops!
    Something went wrong.
    Please try again later.
  • Today FDA has removed the clinical hold on one more gene therapy study, this time for Voyager Therapeutics Inc's (NASDAQ: VYGR) investigational new drug application for VY-HTT01, a gene therapy candidate for Huntington's disease (HD).

  • The agency has confirmed that the company may proceed with its planned Phase 1/2 clinical trial.

  • The decision comes after a comprehensive review of the Chemistry, Manufacturing, and Controls information previously submitted to the FDA.

  • Voyager plans to initiate VYTAL, a Phase 1/2 clinical trial of VY-HTT01, this year.

  • VYTAL Phase 1/2 trial is a dose-escalation study to evaluate the safety and tolerability of VY-HTT01 in patients with early manifest Huntington's disease.

  • Secondary endpoints include disease biomarkers and clinical outcome measures.

  • VY-HTT01 is a gene therapy designed to reduce the expression of huntingtin, thereby altering disease progression.

  • It includes adeno-associated virus capsid and a proprietary transgene that harnesses the canonical RNA interference pathway to knock down levels of HTT mRNA selectively.

  • Earlier today, the FDA removed the clinical hold on uniQure NV's (NASDAQ: QURE) hemophilia B gene therapy program.

  • Price Action: VYGR shares are moving 12.6% higher at $5.25 on the last check Monday.

See more from Benzinga

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