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NFS-02

Development stage: Phase 2
Lead developer: Neurophth
Modality: Gene Therapies
Administration: Ophthalmic

Overview

NFS-02 is a gene therapy product consisting of a recombinant adeno-associated virus serotype 2 containing human mitochondrial ND1 codon optimized gene (rAAV2-ND1). It is designed to treat Leber hereditary optic neuropathy (LHON) associated with mutations in the NADH-dehydrogenase subunit 1 gene (ND1). The therapy is administered as an ophthalmic injection and aims to deliver a codon-optimized copy of ND1 to restore visual function in damaged optic ganglion cells by repairing the mitochondrial respiratory chain.

Other names: recombinant adeno-associated virus serotype 2 containing human mitochondrial ND1 codon optimized generAAV2-ND1rAAV-2-ND1rAAV 2-ND1

Targets

ND1 (Mitochondrial electron transport chain complex I)

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