NFS-02 / Neurophth - LARVOL DELTA

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

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

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

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

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