U.S. FDA approves IND for Reforgene's first-in-class Usher syndrome gene editing medicine

On September 17, 2024, U.S. time, the U.S. Food and Drug Administration (FDA) officially approved the IND application for Reforgene's innovative medicine RM101 for Usher syndrome. This approval marks a milestone breakthrough and advance in the development of the world's first AAV-based gene editing medicine for Usher syndrome.

Retinitis pigmentosa is a group of inherited eye diseases characterized by progressive degeneration of retinal photoreceptor cells, leading to gradual vision loss. Usher syndrome is the most common syndrome associated with retinitis pigmentosa. Usher syndrome is divided into three main types, of which type II is the most prevalent, accounting for about 70% of all Usher cases, with USH2A as the most common causative gene. Mutations in the USH2A gene cause retinitis pigmentosa; patients usually gradually develop night blindness and loss of peripheral vision in adolescence, and may eventually become completely blind.

To date, there is no approved drug or effective treatment for Usher syndrome, which not only severely affects patients' physical and mental health and quality of life, but also imposes a significant burden on families and society, representing a huge unmet clinical need. The development of gene editing technology is expected to reverse this situation and bring new hope to patients. Reforgene's innovative gene editing medicine RM101 induces cells in the patient's retina to produce normal functional proteins through a gene regulation technology route, with the potential for one-time administration and long-lasting effect.

Gene editing technology is an epoch-making tool in the life sciences and a platform-level foundational technology in medicine, bringing a new paradigm to disease treatment. Building on active innovation and deep cultivation of gene editing, Reforgene hopes to use its innovative technologies and drug development platforms to bring breakthrough therapies for diseases that currently cannot be cured, and to bring good news to patients and families suffering from disease. The successful approval of RM101 marks Reforgene's entry into the clinical stage for its gene editing innovative medicines in ophthalmology, laying a solid foundation for future clinical trials and therapeutic applications.

About RM101

RM101 is an innovative medicine developed by Reforgene for retinitis pigmentosa associated with the USH2A gene in Usher syndrome. RM101 is an AAV-based gene editing medicine that specifically targets USH2A RNA, modulates the biological process of alternative splicing, and restores expression of functional protein. Delivered by subretinal injection, RM101 has the potential for one-time administration and long-lasting effect.

About Reforgene

Reforgene Medicine is one of China's leading innovative gene editing drug companies, driven by gene editing technology and dedicated to bringing novel medicines to life. Reforgene has a high-caliber team that applied gene editing to explore the treatment of genetic diseases at an early stage internationally, and has accumulated pioneering achievements in hematology, ophthalmology and other disease areas, with strong technical capabilities in gene editing tool innovation, drug development strategy, novel animal model construction, and multi-level efficacy and safety assessment, operating both in vivo and ex vivo drug development. Reforgene's mission is to deliver accessible, curative medicines for patients with serious diseases through innovative genetic technologies. The company currently has pipeline programs across genetic diseases, complex diseases and oncology. Its innovative beta-thalassemia medicine has achieved globally leading clinical progress, it achieved the world's first cure of an alpha-thalassemia patient, and the IND application for its first-in-class Usher syndrome gene editing medicine has been approved by regulators in both China and the United States.