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RM101

Development stage: Preclinical
Lead developer: Reforgene Medicine
Modality: CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
Administration: Intravitreal

Overview

RM101 is an *in vivo* gene-editing therapeutic candidate being developed by Guangzhou Reforgene Medicine for the treatment of Usher Syndrome, a genetic condition characterized by hearing loss and progressive vision loss due to retinitis pigmentosa. The drug utilizes Reforgene's proprietary gene-editing technology to address the underlying genetic mutations in the eye. As of 2024, RM101 is in the preclinical stage of development, focusing on ophthalmic applications to preserve or restore vision in patients affected by this syndrome.

Targets

USH2A exon 13

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