Reforgene Medicine, a pioneer in gene editing therapy based in Guangzhou, has secured Investigational New Drug (IND) approval from China’s National Medical Products Administration (NMPA) for its flagship candidate, RM-101. This AAV-based gene editing drug is aimed at treating Usher syndrome, a genetic condition that leads to significant vision impairment and often deafness.
Usher syndrome is characterized by retinitis pigmentosa, a group of hereditary diseases that cause the progressive degeneration of retinal photoreceptor cells, resulting in the gradual loss of vision. Type II Usher syndrome, the most prevalent form, accounts for approximately 70% of all Usher cases and is commonly linked to mutations in the USH2A gene. These mutations trigger the onset of night blindness and peripheral visual field loss during adolescence, potentially progressing to complete blindness.
RM-101, an innovative investigational gene therapy, targets the USH2A gene mutation responsible for retinitis pigmentosa in Usher syndrome. Utilizing AAV virus delivery, RM-101 can specifically target USH2A RNA. Administered via subretinal injection, RM-101 infects photoreceptor cells, induces skipping of USH2A exon 13, and produces a truncated but biologically functional Usherin protein. This process helps maintain the normal morphology and function of photoreceptor cells, offering a new therapeutic approach for patients suffering from this debilitating condition.- Flcube.com
