Neurophth and Houd Biological Announce Strategic Collaboration to Co-Develop iPSC-Derived Cell Therapies for Ocular Diseases-VCBeat

BioShell (ID: iBio4P) has learned that today (June 1), Neurophth and Hopstem Biotechnology announced that they will establish a strategic partnership,Both parties will collaborate to develop human iPSC-derived cell products for the treatment of ocular diseases.

It is reported that Neurophth is a leading biopharmaceutical company developing AAV gene therapies for ophthalmic diseases, while Hopstem Biotechnology is a biopharmaceutical company with a leading technology platform for human induced pluripotent stem cell (iPSC) and neural cell differentiation.

This collaboration combines Neurophth’s global experience in the development of gene and cell therapy products and its profound understanding of ocular diseases with Hopstem Biotechnology’s strengths in the GMP manufacturing and quality management systems for human iPSC-derived clinical cell products, holding the promise of delivering next-generation therapies for eye diseases. Under this partnership, Hopstem Biotechnology will receive upfront and milestone payments for the candidate cell product targeting retinal degenerative diseases. Neurophth will secure an exclusive license for this candidate product and assume responsibility for its clinical development and commercialization. Furthermore, Neurophth will be granted corresponding licenses to Hopstem Biotechnology’s patents covering human iPSC reprogramming technology and its GMP-grade human iPSC cell lines, and will pay Hopstem Biotechnology corresponding licensing fees at various stages of product development. Additionally, following the product’s commercial launch, Hopstem Biotechnology will be entitled to receive sales royalties based on product sales.

“This collaboration reflects Neurophth's strategic layout and long-term objectives in developing ophthalmic therapies. We will continue to expand and enrich the Company's pipeline of innovative ophthalmic drug candidates,”Professor Li Bin, Founder and Chairman of Neurophth, said,“Just as in 2008 I believed that gene therapy would become the next-generation treatment in ophthalmology, I believe that induced pluripotent stem cells (iPSCs) will be one of the key trends leading us to define the future. iPSCs possess vast prospects for innovation and hold tremendous potential in areas such as regenerative therapy, disease modeling, and drug screening.”

Dr. Fan Jing, Founder and CEO of Hopstem Biotechnology, stated，“We are highly pleased to partner with Neurophth in this strategic collaboration. The human iPSC cell line we have developed is generated by converting skin tissue from healthy donors into pluripotent stem cells utilizing our proprietary patented technology. Established under GMP-compliant conditions, this iPSC cell line exhibits robust proliferative capacity and the potential to differentiate into any human cell type. Leveraging the manufacturing processes and quality control platform for hiPSC-derived clinical cell products that we have established since 2019, we are positioned to accelerate the development of ophthalmic cell therapies in collaboration with Neurophth, a leading company in ophthalmic treatment. Our shared objective is to deliver safe and effective human iPSC-derived regenerative medicine therapeutics that can, to a certain extent, reverse the progression of ocular diseases and restore patients’ vision.”

“We believe that iPSC technology holds great promise for effectively treating retinal degenerative diseases that cannot be adequately addressed by AAV-mediated gene therapies,” Dr. Yingming Lu, CEO of Neurophth, stated. “Hopstem Biotechnology is one of the most respected pioneers in human iPSC translational medicine. We are highly confident that integrating Hopstem’s technical expertise and deep professional accumulation in iPSCs and directed differentiation with Neurophth’s extensive experience in ophthalmology and drug development will enable us to jointly unlock next-generation innovative gene/cell therapies for patients.”

About Neurophth

Neurophth is China's first gene therapy company dedicated to ophthalmic diseases, with offices in Wuhan, Suzhou, Shanghai, and the United States. As a fully integrated, clinical-stage high-tech enterprise, Neurophth is committed to the global research and development of gene therapies for patients with blindness and other ocular disorders. Leveraging a mature AAV-based ophthalmic gene therapy technology platform and the founding team's decades of in-depth expertise in ophthalmology, Neurophth has established a robust product pipeline. Related research has been published in journals such as *Scientific Reports* (a Nature Portfolio journal), *Ophthalmology*, and *eBioMedicine*, successfully providing proof-of-concept data for retinal gene therapy. The company's lead candidate, NR082 (NFS-01 program, rAAV2-ND4), is a gene therapy for Leber's Hereditary Optic Neuropathy (LHON) caused by ND4 mutations. It has received Orphan Drug Designation from the U.S. FDA and China's first regulatory approval to initiate registration (pivotal) clinical trials for an ophthalmic gene therapy. Our pipeline also includes programs targeting ND1-mediated LHON, autosomal dominant optic atrophy (ADOA), optic nerve protection, vascular optic atrophy, and other preclinical candidates. The company is constructing an internationally compliant GMP commercial-scale manufacturing platform for gene therapies and plans to establish an ophthalmic gene therapy translational center, striving to become a global leader in ophthalmic gene therapy and benefit patients worldwide.

About Hopstem Biotechnology

Hopstem Biotechnology Inc. is one of the first human induced pluripotent stem cell (hiPSC) therapy companies in China. Founded in January 2017, Hopstem was established by neuroscientists and stem cell biologists from Johns Hopkins University in Hangzhou, China, and Baltimore, USA. Hopstem Biotechnology has progressively established a world-leading neural differentiation platform, and holds patents for human induced pluripotent stem cell (hiPSC) reprogramming methods, as well as a high-standard GMP manufacturing and quality management system. Hopstem’s mission is to apply these cutting-edge technologies and platforms to develop innovative cell therapies for the treatment of central nervous system (CNS) diseases and other disorders. Our first clinical product, hNPC01, is a human forebrain neural precursor cell therapy for the treatment of stroke, brain injury, and related conditions. Preliminary studies in rat and monkey pMCAO stroke models demonstrate that the majority of transplanted human forebrain neural precursor cells successfully differentiate into functional neurons and establish new functional connections with distal rat neurons.