Neurophth Therapeutics is a leading clinical-stage gene therapy company with robust ophthalmic pipeline. With subsidiaries in China and USA, Neurophth, a fully integrated company, is striving to discover and develop AAV-delivered gene therapies for patients suffering from vision loss globally. Our most advanced investigational candidate, NFS-01(Esonadogene Imvoparvovec, rAAV2-ND4), in development for treating Leber's Hereditary Optic Neuropathy with ND4 gene mutation (ND4-LHON), has been granted Orphan Drug Designation both by US FDA and EU EMA. The part 1 of its Chinese Phase 1/2/3 registrational clinical trial has reached positive results and the part 2 (Phase 3) will be initiated in Q3 2022. As a gene therapy candidate for global patients, the registrational clinical trial of NFS-01 will be also conducted in US this fall following the IND approval from US FDA in January 2022. Our 2nd advanced investigational candidate, NFS-02 (rAAV2-ND1), in development for treating Leber's Hereditary Optic Neuropathy with ND1 gene mutation (ND1-LHON), has also been granted Orphan Drug Designation by US FDA this January and submitted IND filing to US FDA. Our pipeline also includes:

• Autosomal dominant optic atrophy (ADOA);
• Glaucoma, Non arteritic ischemic optic neuropathy (NAION);
• Diabetic retinopathy (DR), Diabetic macular edema (DME), wet age-related macular edema (w-AMD);
• Bietti's crystalline dystrophy (BCD);
• Corneal dystrophy (FCD);
• X-linked retinitis pigmentosa (XLRP);
• Autosomal dominant retinitis pigmentosa (ADRP);