BioWorld. Link to homepage.

Clarivate
  • BioWorld
  • BioWorld Science
  • BioWorld Asia
  • Data Snapshots
    • Biopharma
    • Medical technology
    • Infographics: Dynamic digital data analysis
    • Index insights
    • NME Digest
  • Special reports
    • Infographics: Dynamic digital data analysis
    • Trump administration impacts
    • Med-tech outlook 2026
    • Under threat: mRNA vaccine research
    • BioWorld at 35
    • Biopharma M&A scorecard
    • Bioworld 2025 review
    • BioWorld MedTech 2025 review
    • BioWorld Science 2025 review
    • Women's health
    • China's GLP-1 landscape
    • PFA re-energizes afib market
    • China CAR T
    • Alzheimer's disease
    • Coronavirus
    • More reports can be found here

BioWorld. Link to homepage.

  • Sign In
  • Sign Out
  • My Account
Subscribe
BioWorld - Wednesday, April 22, 2026
See today's BioWorld
Home » ASH 2022: One-time infusion CSL’s Hemgenix gene therapy shows durable response for hemophilia B
To read the full story, subscribe or sign in.

ASH 2022: One-time infusion CSL’s Hemgenix gene therapy shows durable response for hemophilia B

Dec. 12, 2022
By Tamra Sami
After gaining U.S. FDA priority approval for the first gene therapy to treat hemophilia B, CSL Ltd. reported long-term data from the pivotal HOPE-B trial that showed a single infusion of Hemgenix (etranacogene dezaparvovec-drlb) generated elevated and sustained mean factor IX levels and reduced the rate of annual bleeding. Presented at the American Society of Hematology (ASH) annual meeting on Dec. 10, data showed 24-month results reinforced the safety of treatment, with no serious treatment-related adverse effects.
BioWorld Clinical Conferences American Society of Hematology Hematologic Gene therapy

Popular Stories

  • Today's news in brief

    BioWorld
    BioWorld briefs for April 21, 2026.
  • Man holding raised arm with illustrated overlay of nerves

    Biohaven reports details on discovery of BHV-2100

    BioWorld Science
    Transient receptor potential melastatin 3 (TRPM3) is a calcium-permeable TRP channel that is highly expressed in somatosensory neurons, including nociceptors of...
  • Elderly woman holding illustration of brain with missing puzzle piece

    Meta-analysis: Anti-amyloid Alzheimer’s drugs not ‘clinically meaningful’

    BioWorld
    The ongoing controversy over the effectiveness of anti-amyloid drugs is about to get more heated, after a review of clinical trials showed statistically...
  • New GABA(B) receptor PAMs revealed in Addex Pharma patents

    BioWorld Science
    Addex Pharma SA has disclosed new GABA(B) receptor positive allosteric modulators (PAMs) potentially useful for the treatment of chronic cough, urinary...
  • MMP inhibitors divulged in Accure Therapeutics patent

    BioWorld Science
    Accure Therapeutics has reported new oligopeptide derivatives acting as matrix metalloproteinase-2 (MMP-2) and MMP-9 inhibitors. As such, they are described as...
  • BioWorld
    • Today's news
    • Analysis and data insight
    • Clinical
    • Data Snapshots
    • Deals and M&A
    • Financings
    • Medical technology
    • Newco news
    • Opinion
    • Regulatory
  • BioWorld Science
    • Today's news
    • Biomarkers
    • Cancer
    • Conferences
    • Endocrine/metabolic
    • Immune
    • Infection
    • Neurology/psychiatric
    • NME Digest
    • Patents
  • BioWorld Asia
    • Today's news
    • Analysis and data insight
    • Australia
    • China
    • Clinical
    • Deals and M&A
    • Financings
    • Newco news
    • Regulatory
    • Science
  • More
    • About
    • Advertise with BioWorld
    • Archives
    • Article reprints and permissions
    • Contact us
    • Cookie policy
    • Copyright notice
    • Data methodology
    • Infographics: Dynamic digital data analysis
    • Index insights
    • Podcasts
    • Privacy policy
    • Share your news with BioWorld
    • Staff
    • Terms of use
    • Topic alerts
Follow Us

Copyright ©2026. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing