Optic Atrophy Gene Therapy Cleared for Trials in Australia


NFS-05 is an ophthalmic injection that uses an AAV vector to deliver the OPA1 gene to retinal ganglion cells.

Neurophth’s NFS-05 gene therapy has been cleared by the Australian Therapeutic Goods Administration for clinical trials in people with Autosomal Dominant Optic Atrophy (ADOA).1

"Neurophth's NFS-05 marks our third drug to receive clinical trial approval and our inaugural approval in Australia," Professor Li Bin, MD, PhD, founder, chairman, and chief executive officer, Neurophth, said in a statement.1 "This achievement underscores Neurophth's robust R&D capabilities and our unwavering commitment to global outreach. We remain dedicated to harnessing our technical platform, broadening our product pipeline, and diligently expanding beyond China to deliver innovative medical solutions to patients globally."

Around 80% of ADOA cases are caused by mutations in the OPA1 gene, the protein reduction of which leads to mitochondrial fragmentation, and increased instability of the mitochondrial respiratory chain complex. This leads toretinal ganglion cell (RGC) death and optic nerve atrophy, with patients experiencing bilateral, slowly progressing visual impairment, temporal pallor of the optic disc, central visual field defects, and abnormalities in color vision. NFS-05 is an ophthalmic injection that delivers an RGC-targeted, adeno-associated vector with the OPA1 gene into the vitreous cavity to restore gene expression.

READ MORE: Ocugen’s Gene-Agnostic Gene Therapy OCU400 Demonstrates Improvement or Stabilization in Retinitis Pigmentosa, Leber Congenital Amaurosis

"Existing clinical interventions for ADOA, including measures to enhance circulation and support nerve health, offer limited benefit. With NFS-05, Neurophth endeavors to target the underlying cause of the disease using gene therapy, aiming for enhanced visual outcomes for patients,” Xiaoning Guo, PhD, chief medical officer, Neurophth, added.1 “We are committed to advancing international multi-center clinical trials for NFS-05, with the goal of rapidly delivering a safe and efficacious treatment to patients."

Last month, Neurophth also announced that it had dosed the first patient in a multiregional phase 1/2 clinical trial (NCT05820152) evaluating Neurophth Therapeutics’ NFS-02 (rAAV2-ND1), an investigational gene therapy intended to treat Leber hereditary optic neuropathy associated with mutations in the NADH-dehydrogenase subunit 1 gene (LHON-ND1).2

NFS-02 is also an ophthalmic injection that delivers a codon-optimized copy of ND1 via an AAV2 vector.The study, which is being conducted in both the United States and China, is led by Jia Qu, MD, and Rong Zhou, MD, professors at the Eye Hospital of Wenzhou Medical University. Several patients have already received NFS-02 in a separate investigator-initiated trial in China.

“Today’s milestone is a significant step forward,” Li said in an August statement.2 “NFS-02 is Neurophth’s second gene therapy to enter clinical trials, demonstrating the company's strong operations. NFS-02 is currently the only ND1-LHON treatment in development worldwide. In response to patients’ expectations, we are committed to conduct global multiregion, multicenter clinical trials to ensure that it’s on the path towards early drug approval and commercialization in the future.”

1. Neurophth secures Australian approval for ophthalmic gene therapy. News release. Neurophth. September 6, 2023. Accessed September 25, 2023. https://www.prnewswire.com/news-releases/neurophth-secures-australian-approval-for-ophthalmic-gene-therapy-301918750.html
2. Neurophth announces first patient dosed in phase I/II clinical trial of second gene therapy. News release. Neurophth Therapeutics, Inc. August 17, 2023. Accessed September 25, 2023. https://www.neurophth.com/en/NewsD-393.html
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