Neurophth Biotechnology Ltd, a leading gene therapy specialist based in China, has announced that it has received approval from the National Medical Products Administration (NMPA) to initiate a Sino-US multi-center Phase I/II study. The study will assess the safety, efficacy, and tolerability of its gene therapy NFS-02 (rAAV2-ND1) for ND1 mutation-induced Leber hereditary optic neuropathy (ND1-LHON). This development marks a significant step forward for the company in its pursuit of treatments for genetic eye diseases.
Background on NFS-02 and Orphan Drug Designation
The investigational in vivo gene therapy, NFS-02, previously received an orphan drug designation (ODD) in the US in January of the previous year. This designation recognizes the drug’s potential to treat rare conditions and offers development incentives for addressing unmet medical needs.
Neurophth’s Core Product and Clinical Progress
Neurophth’s first core product, NR082, which also holds ODD status from both the US and EU authorities, has achieved a full enrollment of patients in a Phase III study in China and has initiated trials in the US. NR082 targets a different genetic eye condition, highlighting the company’s broad pipeline in gene therapy for ophthalmic diseases.
Pre-Clinical Pipeline and Future Prospects
Beyond its clinical programs, Neurophth has a robust pre-clinical drug pipeline targeting various eye conditions, including autosomal dominant optic atrophy, optical neuroprotection, and vascular retinopathy. These pipeline developments indicate the company’s commitment to advancing novel treatments for a range of vision-threatening diseases.
Conclusion
The NMPA’s approval for Neurophth Biotechnology’s Phase I/II study of NFS-02 is a significant milestone, reflecting the company’s progress in gene therapy research and development. With a strong pipeline and international clinical trials underway, Neurophth is well-positioned to contribute to the field of genetic ophthalmology.-Fineline Info & Tech
