BioWorld. Link to homepage.

Clarivate
  • BioWorld
  • BioWorld MedTech
  • BioWorld Asia
  • BioWorld Science
  • Data Snapshots
    • BioWorld
    • BioWorld MedTech
    • Infographics: Dynamic digital data analysis
    • Index insights
    • NME Digest
  • Special reports
    • Infographics: Dynamic digital data analysis
    • Trump administration impacts
    • 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 - Friday, January 9, 2026
See today's BioWorld
Home » Biomarin sees durability in hemophilia A gene therapy, gives phase III data peek
To read the full story, subscribe or sign in.

Biomarin sees durability in hemophilia A gene therapy, gives phase III data peek

May 29, 2019
By Michael Fitzhugh
New data on Biomarin Pharmaceutical Inc.'s investigational treatment for severe hemophilia A, valoctocogene roxaparvovec (valrox), sparked mild debate among analysts who expressed appreciation for the gene therapy's apparent durability but uncertainty regarding interim data from a phase III test of the medicine. But overall, it "at least met – and in some cases exceeded – the majority of investors' expectations," J.P. Morgan analyst Cory Kasimov said. Though the market rendered its own verdict, driving company shares (NASDAQ:BMRN) 5.1% lower to $84.50 by Tuesday's close, the program seemed poised to retain significant interest going forward.
BioWorld

Popular Stories

  • Today's news in brief

    BioWorld
    BioWorld briefs for Jan 8, 2025.
  • Today's news in brief

    BioWorld MedTech
    BioWorld MedTech briefs for Jan. 8, 2026.
  • 3D rendering of antibody drug conjugated with cytotoxic payload

    ADCs’ breakout 2025 and their still-unfinished potential

    BioWorld Science
    Over the course of the year, and continuing into the latest scientific meetings, an extraordinary breadth of new antibody-drug conjugate (ADC) designs was...
  • CAR T cell attacking cancer cells

    Development and characterization of anti-CD19 in vivo CAR T therapy

    BioWorld Science
    Shenzhen Grit Biotechnology Co. Ltd. and Shanghai Vitalgen Biopharma Co. Ltd. recently presented their work to develop and evaluate a novel anti-CD19 in vivo CAR...
  • Preclinical results of anti-ADAMTS13 antibody in acquired von Willebrand syndrome

    BioWorld Science
    The acquired von Willebrand syndrome (AVWS) is a rare bleeding disorder with laboratory findings similar to those of inherited von Willebrand disease. Researchers...
  • BioWorld
    • Today's news
    • Analysis and data insight
    • Clinical
    • Data Snapshots
    • Deals and M&A
    • Financings
    • Newco news
    • Opinion
    • Regulatory
    • Science
  • BioWorld MedTech
    • Today's news
    • Clinical
    • Data Snapshots
    • Deals and M&A
    • Financings
    • Newco news
    • Opinion
    • Regulatory
    • Science
  • BioWorld Asia
    • Today's news
    • Analysis and data insight
    • Australia
    • China
    • Clinical
    • Deals and M&A
    • Financings
    • Newco news
    • Regulatory
    • Science
  • BioWorld Science
    • Today's news
    • Biomarkers
    • Cancer
    • Conferences
    • Endocrine/Metabolic
    • Immune
    • Infection
    • Neurology/Psychiatric
    • NME Digest
    • Patents
  • 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