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 - Friday, April 10, 2026
Breaking News: Best of BioWorld Science: Q1See today's BioWorld Science
Home » Neurophth cleared by FDA to enter clinic with AAV-ND1 gene therapy for LHON
To read the full story, subscribe or sign in.
Ocular

Neurophth cleared by FDA to enter clinic with AAV-ND1 gene therapy for LHON

Dec. 20, 2022
Neurophth Therapeutics Inc. has received FDA clearance of its IND application for the in vivo gene replacement therapy NFS-02, a novel recombinant adeno-associated viral serotype 2 vector (rAAV2) containing a codon-optimized NADH-dehydrogenase subunit 1 (ND1) gene, for the treatment of Leber hereditary optic neuropathy (LHON) associated with ND1 mutation.
BioWorld Science Ocular Gene therapy FDA IND

Popular Stories

  • Today's news in brief

    BioWorld
    BioWorld briefs for April 9, 2026.
  • Hengrui discovers new Nav1.8 blockers

    BioWorld Science
    Researchers from Jiangsu Hengrui Pharmaceuticals Co. Ltd. and Shanghai Hengrui Pharmaceutical Co. Ltd. have patented new sodium channel protein type 10 subunit α...
  • Cancer and blood cells

    Hematopoietic stem cell research points to leukemia’s early roots

    BioWorld Science
    Hematopoietic stem cell (HSC) research over the past century has shown that leukemia may be driven by an invisible hand of inflammation. The bone marrow and...
  • News in brief

    BioWorld Asia
    BioWorld Asia briefs for April 7, 2026
  • Connecting puzzle pieces

    Korsana boosts Alzheimer’s work with reverse merger, $380M financing

    BioWorld Science
    Just over a month after emerging from stealth and disclosing a $150 million series A, Korsana Biosciences Inc. is making the leap to the public market via a...
  • 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