Ruijian Medicine's Breakthrough Ophthalmic Therapy NouvSight001 Receives FDA Orphan Drug Designation for Retinitis Pigmentosa

On March 20, 2024, NouvSight001, a breakthrough ophthalmic product by Wuhan iRegene Pharmaceutical Technology Co., Ltd. (iRegene), was granted Orphan Drug Designation (ODD) by the U.S. FDA. Targeting "a series of retinitis pigmentosa indications," this product is the world’s first universal ophthalmic cell therapy derived from induced pluripotent stem cells (iPSC), developed by the iRegene team using its unique "AI + chemical induction" platform. It also represents a globally pioneering breakthrough in ophthalmic treatment.Retinitis Pigmentosa (RP) is a hereditary ophthalmic disease that can ultimately lead to blindness. Symptoms include night blindness and peripheral vision loss, which gradually worsens, potentially leading to tunnel vision (narrowed field of vision) or even complete blindness. RP can be caused by mutations in any of more than 50 related genes, with the pathogenesis mainly attributed to progressive loss of rod cells and cone cell degeneration in the retina. Currently, there is no effective clinical treatment for RP.

This ophthalmic product NouvSight001F by the United StatesDAThe granting of orphan drug designation marks a significant milestone in the development process of iRegene. Regarding this,iRegene CDr. MO Cai MengFDA's orphan drug designation for iRegene's ophthalmic product NouvSight001 demonstrates the company's strategic foresight in early pipeline planning and indication selection strategy. It also recognizes the product's uniqueness in the field of retinal disease treatment and its superior therapeutic mechanism. This orphan drug designation will significantly accelerate the product’s subsequent commercialization process. iRegene will also gain market exclusivity upon the product’s market launch, enhancing the commercial value of the pipeline. The company will continue to push forward with the registration and clinical trials of the product to benefit patients worldwide as soon as possible.Emmanuel Montet, SVP of International Affairs at iRegeneThis ODD award demonstrates that iRegene's innovative spirit and relentless efforts have once again achieved a significant milestone. It is the team's rapid learning ability, hard work, and dedication that have brought iRegene to this critical moment. The team's drive for excellence has further strengthened iRegene's determination to address the challenges faced by patients with retinitis pigmentosa.

iRegene, as one of the earliest biotech companies globally to apply "AI + chemical induction" to the specific functional modification of cells, has established a rich pipeline of universal iPSC products on this unique platform. It aims to develop universal cell therapy products and provide new treatment options for currently "incurable" diseases such as neurodegenerative disorders represented by Parkinson’s disease and blindness. Relying on its self-developed platform based on "AI + chemical induction" for precise functional remodeling of cells, iRegene determines the induction protocols for derivative cells through accurate differential analysis, achieving efficient transformation of induced pluripotent stem cells (iPSCs) and ensuring their drug applicability. The advantages of the "AI + chemical induction" platform have been validated in clinical trials. NouvNeu001, the first universal cell therapy product from iRegene and the world's first universal chemically-induced product for Parkinson’s disease, was approved to enter combined Phase I-II clinical trials in August 2023. The first patient was dosed in January 2024, and promising efficacy and safety data have already been obtained in the ongoing registrational clinical trial.