China approves IND for Reforgene's first-in-class Usher syndrome gene editing medicine

The IND application for RM101, Reforgene's gene editing medicine for Usher syndrome developed independently by the company, obtained implied clearance from the Center for Drug Evaluation (CDE) of the National Medical Products Administration on October 11, 2024. This marks the upcoming entry into the clinical stage in China of the world's first Usher syndrome gene editing medicine, bringing new hope to Chinese patients. It is worth noting that RM101 had already received IND approval from the U.S. Food and Drug Administration (FDA) on September 17, U.S. time. To date, Reforgene has achieved dual IND approval of an innovative Usher syndrome gene editing medicine in both China and the United States.

Retinitis pigmentosa is a group of severe 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. Patients with Usher syndrome experience progressive loss of visual function, starting with reduced night vision and gradually developing into narrowed visual fields, reduced central vision and even blindness, generally accompanied by non-degenerative hearing impairment.

At present, there is no approved drug or treatment for Usher syndrome, representing a huge unmet clinical need. Gene editing therapy is expected to change this situation. RM101, the gene editing medicine independently developed by Reforgene, is expected to induce functional protein expression in the patient's retinal photoreceptor cells through a one-time subretinal injection, ultimately improving the patient's vision.

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.