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 - Thursday, March 19, 2026
See today's BioWorld
Home » Regenxbio wrestling holds on MPS I, II gene therapy trials
To read the full story, subscribe or sign in.

Regenxbio wrestling holds on MPS I, II gene therapy trials

Jan. 28, 2026
By Randy Osborne
No Comments
With the PDUFA date fast approaching for Regenxbio Inc.’s gene therapy RGX-121, the U.S. FDA placed the drug on clinical hold along with another, RGX-111, after preliminary analysis of a single case of neoplasm (specifically, an intraventricular central nervous system tumor) in a participant treated in the phase I/II study with the latter treatment.
BioWorld Clinical Endocrine/metabolic Gene therapy FDA

Popular Stories

  • Today's news in brief

    BioWorld

    BioWorld briefs for March 19, 2026.

  • Art concept for targeting the brain

    Precision psychiatry beyond, or before, biomarkers

    BioWorld
    There is broad agreement that psychiatric diagnoses in their current form are not reflective of any underlying biology, and that this is one of the things...
  • Illustration of a motor neuron

    Keros reports beneficial effects of RKER-065 in ALS model

    BioWorld Science
    Keros Therapeutics Inc. has presented data regarding their activin receptor ligand trap, RKER-065, for the inhibition of the activin/myostatin signaling axis.
  • News in brief

    BioWorld Asia
    BioWorld Asia briefs for March 17, 2026
  • First-in-class POLG activator restores mtDNA across mutations

    BioWorld Science
    Researchers from Pretzel Therapeutics Inc. presented preclinical data of PX-578, a first-in-class POLG activator aimed at restoring mtDNA replication and...
  • 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