China-based gene therapy specialist Neurophth Biotechnology Ltd has announced that it has received clinical trial approval from the Therapeutic Goods Administration of Australia for its candidate drug NFS-05. The therapy is set to be assessed as a treatment for autosomal dominant optic atrophy (ADOA), an inherited optic neuropathy.
Understanding Autosomal Dominant Optic Atrophy (ADOA)
ADOA is caused by mutations in the OPA1 gene in approximately 80% of cases. The decline in the function of the OPA1 protein results in mitochondrial fragmentation and increased instability of mitochondrial respiratory chain complexes, damaging mitochondrial function and leading to retinal ganglion cell (RGC) apoptosis and optic nerve atrophy. Patients with ADOA typically experience a bilateral, symmetrical, and slowly progressive decrease in vision, pale bilateral temporal optic discs, central visual field defects, and color vision impairment.
NFS-05: A Pioneering Gene Therapy Approach
The in-house developed NFS-05 ophthalmic injection represents a gene therapy strategy where an adeno-associated virus (AAV) vector carrying the OPA1 gene is injected into the vitreous cavity. The virus infects RGC cells and expresses the OPA1 protein, thereby repairing mitochondrial function and potentially reversing the effects of ADOA.-Fineline Info & Tech
