Neurophth's Gene Therapy NFS-05 for ADOA Receives Clinical Trial Approval in Australia
Neurophth
Ophthalmic Gene Drug Developer
Australian Therapeutic Goods Administration
The Australian Therapeutic Goods Administration is an Australian government agency responsible for evaluating, assessing, and monitoring products defined as therapeutic goods. We regulate medicines, medical devices, and biological products to help Australians stay healthy and safe.
Today (September 6), Neurophth Biotechnology Ltd. (hereinafter referred to as "Neurophth") announced that its candidate drug NFS-05, for the treatment of Autosomal Dominant Optic Atrophy (ADOA), has completed the Therapeutic Goods Administration (TGA) clinical trial registration in Australia and has been approved to commence clinical trials.
Autosomal Dominant Optic Atrophy (ADOA) is an autosomal dominant optic neuropathy, with approximately 80% of ADOA cases caused by mutations in the OPA1 gene. Reduced function of the OPA1 protein leads to mitochondrial fragmentation and increased instability of mitochondrial respiratory chain complexes, thereby impairing mitochondrial function, ultimately resulting in RGC apoptosis and optic nerve atrophy. Clinically, patients exhibit roughly symmetrical, slowly progressive bilateral vision loss, bilateral temporal disc pallor, central scotoma, and dyschromatopsia.
Currently, there are no effective clinical treatments available for ADOA. NFS-05 ophthalmic injection, independently developed by Neurophth, utilizes a gene therapy approach. It involves injecting an AAV vector carrying the OPA1 gene into the vitreous cavity, where the virus infects RGC cells and expresses the OPA1 protein, thereby restoring mitochondrial function.
Editor: Muyan
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