NFS-02
/ Neurophth
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October 24, 2025
Development of Two In Vitro ND1-LHON Models for Evaluating Gene Therapy Efficacy.
(PubMed, Invest Ophthalmol Vis Sci)
- "The rAAV-ND1 gene therapy effectively restored mitochondrial function, highlighting its potential as a treatment for LHON caused by ND1 mutations. These findings underscore the value of in vitro systems for evaluating therapies when robust animal models are unavailable."
Journal • Preclinical • Gene Therapies • Inherited Retinal Dystrophy • Leber Hereditary Optic Neuropathy • Metabolic Disorders • Ocular Inflammation • Ophthalmology • Optic Neuritis
May 08, 2025
NFS-02 Nucleic acid medicine and cellular medicine in the treatment of neurological diseases and the leading edge of research
(JSNE 2025)
- No abstract available
CNS Disorders
September 20, 2024
Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations
(clinicaltrials.gov)
- P1/2 | N=11 | Terminated | Sponsor: Neurophth Therapeutics Inc | N=18 ➔ 11 | Trial completion date: Dec 2029 ➔ Jun 2024 | Recruiting ➔ Terminated | Trial primary completion date: Dec 2024 ➔ Jun 2024; Due to the sponsor circumstances and external reasons
Enrollment change • Gene therapy • Trial completion date • Trial primary completion date • Trial termination • Gene Therapies • Inherited Retinal Dystrophy • Leber Hereditary Optic Neuropathy • Ocular Inflammation • Ophthalmology • Optic Neuritis • Pain
September 13, 2023
Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations
(clinicaltrials.gov)
- P1/2 | N=18 | Recruiting | Sponsor: Neurophth Therapeutics Inc | Not yet recruiting ➔ Recruiting
Enrollment open • Gene therapy • Gene Therapies • Inherited Retinal Dystrophy • Leber Hereditary Optic Neuropathy • Ocular Inflammation • Ophthalmology • Optic Neuritis • Pain • IFNG
April 19, 2023
Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations
(clinicaltrials.gov)
- P1/2 | N=18 | Not yet recruiting | Sponsor: Neurophth Therapeutics Inc
Gene therapy • New P1/2 trial • Gene Therapies • Inherited Retinal Dystrophy • Leber Hereditary Optic Neuropathy • Ocular Inflammation • Ophthalmology • Optic Neuritis • Pain • IFNG
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