Global First! RuiFeng Biotech’s Self-Developed Ophthalmic Gene Editing Drug RM-101 Approved for Domestic Clinical Trials

RuiFeng Biotech’s self-developed Usher syndrome gene-editing drug RM-101 received implied approval from the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) on October 11, 2024. This marks that RuiFeng Biotech’s first global Usher syndrome gene-editing drug is about to enter the clinical phase in China, bringing new hope to patients in the country.

Notably, RM-101 also received IND approval from the U.S. Food and Drug Administration (FDA) on September 17 (U.S. local time). To date, RuiFeng Biotech has achieved “dual submission and dual approval” of INDs for its innovative Usher syndrome gene-editing therapy 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, which leads to the gradual loss of vision. Usher syndrome is the most common syndromic condition associated with retinitis pigmentosa. Usher syndrome is mainly divided into three types; Type II is the most prevalent, accounting for approximately 70% of all Usher cases, with its most common causative gene being USH2A. Patients with Usher syndrome experience progressive loss of visual function—initially presenting with reduced night vision, then gradually progressing to narrowed visual fields, loss of central vision, and ultimately blindness—typically accompanied by non-degenerative hearing abnormalities.

At present, there are no approved drugs or treatments for Usher syndrome, resulting in substantial unmet clinical needs. Gene-editing therapy is expected to help change this landscape. RuiFeng Biotech’s RM-101 is expected to induce the production of functional proteins in patients’ 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 RuiFeng Biotech for USH2A gene-related retinitis pigmentosa associated with Usher syndrome. RM-101 is an AAV-based gene-editing therapy that can specifically target USH2A RNA, regulate the biology of alternative splicing, and restore the expression of functional, normally functioning proteins. Administered via subretinal injection, RM-101 is expected to achieve one-time dosing with long-term therapeutic benefit.