With 41st Annual J.P. Morgan Healthcare Conference 2023 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 clinicaltrial has reached positive results and the first patient of part
2 (Phase 3) has been dosed in September 2022. As a gene therapy candidate for
global patients, the registrational clinicaltrial of NFS-01 has also been initiated in US following the
IND approval from US FDA in January 2022 and anticipated first patient dosed in
January 2023. 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 this January by FDA and received IND approval this December from
US FDA. Our pipeline also includes:
· Autosomal dominant optic atrophy (ADOA);
· Glaucoma;
· Diabetic Retinopathy (DR);
· Geographic Atrophy (GA);
· 4 other preclinical candidates.
Additionally, Neurophth owns in-house gene therapy cGMP manufacturing facility compliant with the cGMP requirements of FDA, EMA and NMPA at Suzhou certificated through NMPA and FDA INDs. The facility has started 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 (www.neurophth.com), please contact Su Zhang, our CFO or Luke Li , our Head of BD and Commercial. We look forward to seeing you at JPM2023 .