RM-101

RM-101 is an adeno-associated virus (AAV)-based gene therapy developed by Guangzhou Ruifeng Biological Technology. It is designed to act as a stimulator of the HBG gene and a modulator of usherin, targeting both the HBG1 gene and usherin protein. The primary therapeutic focus for RM-101 is on genetic disorders affecting the nervous system and eyes, particularly Usher syndrome type 2 and related Usher syndromes. The mechanism involves stimulating fetal hemoglobin (HBG) expression and modulating usherin function, which may address underlying genetic defects in these conditions[1]. Early clinical development has also included evaluation in retinitis pigmentosa[3].