Neurophth Therapeutics develops AAV-based gene therapies for ophthalmic diseases, including advanced investigational programs such as NR082 for ND4-LHON. Its AAV platform has been validated by data from an investigator-initiated retinal gene therapy study. NR082 has received orphan drug designation from both the U.S. FDA and the EMA. The company’s pipeline also includes programs for autosomal dominant optic atrophy, optic neuroprotection, vascular retinopathy and other preclinical candidates. Neurophth has subsidiaries in China (Wuhan, Shanghai and Suzhou) and the U.S. (San Diego, CA) and is led by founder, chairman and CEO Bin Li. The firm intends to use the new proceeds to accelerate clinical trials, enhance R&D capabilities and expand its pipeline. Neurophth Therapeutics develops gene therapies targeting ophthalmic diseases, with its lead investigational candidate NR082 (NFS-01, rAAV2-ND4) for ND4-mediated LHON. NR082 has orphan drug designation from the U.S. FDA and its IND for a Phase 1/2/3 trial was approved by China NMPA in March 2021, with the first patient dosed in June 2021. The company’s pipeline includes ND1-mediated LHON, autosomal dominant optic atrophy, optic neuroprotection (e.g., glaucoma), vascular retinopathy (e.g., diabetic retinopathy) and five other preclinical candidates. Neurophth has initiated scale-up of in-house manufacturing using single-use technologies at its Suzhou facilities to support future commercial demand. The company intends to use newly raised capital to accelerate growth, advance its lead LHON program ex-China, enrich its R&D pipeline through business development, and continue expanding its international-standard gene therapy manufacturing platform. Neurophth is a fully integrated genetic medicines company focused on AAV-delivered gene therapies for ocular diseases. Its lead investigational candidate is NR082 (rAAV2-ND4) for ND4-mediated Leber hereditary optic neuropathy (LHON), which received U.S. FDA orphan designation in Q3 2020. The company expects to advance NR082 toward IND submission and to initiate clinical trials in the first half of 2021. Neurophth is developing a pipeline of 10 gene therapy product candidates addressing both rare and common ocular diseases, including ND1-mediated LHON, autosomal dominant optic atrophy, glaucoma, and diabetic macular edema. It is building a commercially scalable GMP manufacturing facility in Suzhou and scaling up in-house manufacturing using single-use technologies, and plans to establish translational research centers. Headquartered in Wuhan with subsidiaries in Shanghai, Suzhou, and the US, Neurophth closed a Series A in early 2020 to initiate facility construction and accelerate nonclinical studies.

Total raised

$217M

Funding rounds

3

Latest round

Series C

Latest activity

Aug 2023