The 3rd BIO International Convention 2022 is Approaching

09 June 2022 | Thursday | News

With the 3rd BIO International Convention 2022 soon approaching, Neurophth Therapeutics is looking forward to discussing collaboration opportunities in gene therapy for both rare and common ocular diseases.

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);
Additionally, Neurophth owns in-house gene therapy GMP manufacturing facility in accordance with international quality standards at Suzhou certificated through NMPA and FDA INDs that came into operation since Sept. 2021. This will support our supply strategy and realize our costs of goods management strategy. To learn more about us or for collaboration opportunities with Neurophth (, please contact Jennifer Li, our CSO or Luke Li, our head of BD and Commercial. We look forward to seeing you at BIO2022.


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