Neurophth Biotechnology Ltd., a fully-integrated genetic medicines company developing AAV-mediated gene therapies for the treatment of ocular diseases, and Hopstem Biotechnology, the leading human induced pluripotent stem cell (hiPSC) and neural differentiation technology platform company, announced a strategic partnership aiming to provide human iPSC-derived cell therapy for ocular diseases.

The partnership leverages Neurophth's experience in global gene/cell therapy drugs development and understanding of ophthalmic diseases to complement Hopstem's expertise in GMP manufacturing and quality assurance of iPSC-derived clinical cell products to provide next-generation ocular treatments. Under the terms of agreement, Hopstem Biotechnology will receive upfront and milestone payments for the development of candidate cell product for agreed retinal degenerative disorder. Neurophth will have the option to license the candidate product and will be responsible for development and commercialization of the licensed product. In addition, Neurophth agreed to license Hopstem's iPSC reprogramming patent and GMP iPSC line with additional payments to Hopstem at different product development stages. According to the agreement, Hopstem will also share part of the product sales.

"This collaboration exemplifies Neurophth's long-term commitment to advancing the field of ophthalmic treatment as we continue to expand and progress our innovative pipeline of ocular candidates," said Bin Li, M.D., Ph.D., Founder and Chairman of Neurophth Therapeutics. "Combining the methods of stem cell technology, induced pluripotent stem cells is a promising technology that can offer an extraordinary potential for regenerative therapy, disease modeling and drug screening."

"We are very excited by this partnership with Neurophth. The human induced pluripotent stem cell (hiPSC) line we developed is made by transforming the skin of a healthy donor into stem cells that are capable of multiplying and becoming any type of cell in the human body, meeting GMP requirements. Thanks to our iPSC-derived clinical cell product manufactory and quality platform developed since 2019, we are able to speed up ocular cell product development with Neurophth, the leading ocular therapy company in the field. Together, our aim is to offer safe and effective regenerative medicine with hiPSC-derived cells for reversing the progression of ocular diseases and restoring vision for patients," Jing Fan, Ph.D., Founder and CEO of Hopstem Biotechnology.

"iPSCs holds the promise for treatment of retinal degenerative disorders where AAV-mediated gene therapy is unreachable," said Alvin Luk, Ph.D., M.B.A., CEO at Neurophth. "Hopstem is one of the most respected pioneers in the field of iPSC translational medicine. We are confident that their technology and expertise, combined with Neurophth's deep knowledge in ophthalmology and drug development, has the potential to unlock future generations of gene/cell therapy treatments for patients."

About Neurophth

Neurophth is China's first gene therapy company for ophthalmic diseases.  Headquartered in Wuhan with subsidiaries in Shanghai, Suzhou, and US, Neurophth, a fully integrated company, is striving to discover and develop gene therapies for patients suffering from blindness and other eye diseases globally. Our validated AAV platform which has been published in Nature - Scientific Reports, Ophthalmology, and EBioMedicine, has successfully delivered proof-of-concept data with investigational gene therapies in the retina. Our most advanced investigational candidate, NR082 (NFS-01 project, rAAV2-ND4), in development for the treatment of ND4-mediated Leber hereditary optic neuropathy (LHON), has granted orphan designation by the U.S FDA and its IND has also been approved by China NMPA. The pipeline also includes ND1-mediated LHON, autosomal dominant optic atrophy, optic neuroprotection (e.g., glaucoma), vascular retinopathy (e.g., diabetic macular edema and wet age-related macular degeneration), and five other preclinical candidates.  Neurophth has initiated the scaling up in-house manufacturing process in single-use technologies to support future commercial demand at the Suzhou facility. 

About Hopstem

Hopstem Biotechnology is one of the first few iPSC cell therapy companies in China. The company was founded in January 2017 in Hangzhou (China) and Baltimore (US) by neuroscientists and stem-cell biologists from Johns Hopkins University. Hopstem has established a world-leading neural differentiation platform as well as patented iPSC reprogramming method and high standard GMP manufactory and quality system. The mission of Hopstem is to apply these cutting-edge technologies to develop innovative cell therapies for CNS and other disorders. Our leading clinical product, hNPC01, is a human forebrain neural progenitor cell product for stroke and traumatic brain injuries, etc. Preliminary studies in rat and monkey pMCAO stroke models have suggested that majority of those transplanted hNPCs differentiated into functional neural cells and formed significant new connections with the rat neurons in distal regions.