Warm Congratulations to Reforgene Medicine on Clinical Trial Approval in China for RM-101, the World’s First Self-Developed Ophthalmic Gene-Editing Therapy

The Investigational New Drug (IND) application for RM-101, a gene-editing drug for Usher syndrome independently developed by Reforgene Medicine, received implied approval from the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) on October 11, 2024. This milestone marks that Reforgene Medicine’s RM-101, the world’s first gene-editing drug for Usher syndrome, is set to enter the clinical stage in China, bringing new hope to Chinese patients.

Notably, RM-101 received IND clearance from the U.S. Food and Drug Administration (FDA) on September 17, U.S. time. With this achievement, Reforgene Medicine has successfully obtained IND approvals for its innovative gene-editing drug for Usher syndrome in both China and the United States.

Retinitis pigmentosa is a group of severe inherited ophthalmic diseases primarily characterized by the 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 mainly classified into three types, among which Type II is the most prevalent, accounting for approximately 70% of all Usher cases, with USH2A being the most common causative gene. Patients with Usher syndrome experience progressive loss of visual function, initially presenting as impaired night vision and gradually progressing to visual field constriction, central vision loss, and even blindness, generally accompanied by non-degenerative hearing abnormalities.

Currently, there are no approved drugs or therapies for Usher syndrome, representing a significant unmet clinical need. Gene-editing therapy has the potential to change this treatment landscape. RM-101, a gene-editing drug independently developed by Reforgene Medicine, is expected to induce the production of functional protein in retinal photoreceptor cells through a one-time subretinal injection, ultimately improving patients’ vision.

About RM-101

RM-101 is an innovative drug product developed by Reforgene Medicine for Usher syndrome and USH2A gene-associated retinitis pigmentosa. RM-101 is an AAV-based gene-editing drug that specifically targets USH2A RNA, regulates the biological process of alternative splicing, and induces the restoration of functional protein expression.

Administered via subretinal injection, RM-101 is expected to achieve long-term efficacy through a single administration.