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
    • Ebola outbreak
    • Hantavirus
    • 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 - Sunday, May 31, 2026
See today's BioWorld
Home » Uniqure reassures with full-year low-dose data in HD; efficacy ahead with gene therapy?
To read the full story, subscribe or sign in.

Uniqure reassures with full-year low-dose data in HD; efficacy ahead with gene therapy?

June 23, 2022
By Randy Osborne
Uniqure NV rolled out stock-pleasing safety and biomarker data from 10 patients enrolled in the low-dose cohort of the ongoing phase I/II trial with AMT-130 for the treatment of Huntington’s disease (HD), but investors must wait for details on MRI scans. “We have communicated that we will discuss the MRI findings in the middle of next year,” CEO Matt Kapusta told investors during a conference call. “That’s all I can say.”
BioWorld Clinical Neurology/psychiatric Gene therapy

Popular Stories

  • Today's news in brief

    BioWorld
    BioWorld briefs for May 29, 2026.
  • Floating antibody drug conjugates

    TROP2 ADCs progress into first-line for lung, breast cancers

    BioWorld
    Multiple updates on TROP2-directed antibody-drug conjugates (ADCs) for lung and breast cancers highlight both progress made and opportunities for improvement for...
  • Illustration of antibodies and viral infection

    New evidence links autoimmunity to long COVID symptoms

    BioWorld
    Recent findings are reshaping current understanding of the post-infection landscape of SARS-CoV-2. Although previous studies had already suggested that...
  • Digital brain and silhouette

    Will Ovid’s golden age of KCC2 metamorph CNS?

    BioWorld
    The “deep dive” April 8 by Ovid Therapeutics Inc. into potassium-chloride co-transporter 2 (KCC2) research included company officials and independent experts in...
  • U.S. Capitol building

    BMS-Hengrui deal strikes panic in Washington

    BioWorld
    On the heels of the multibillion-dollar licensing deal between Bristol Myers Squibb Co. and Jiangsu Hengrui Pharmaceuticals Co. Ltd., Chairman John Moolenaar...
  • 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