Drug intelligence / Profile preview

RG1501

Development stage
Preclinical
Lead developer
RheumaGen
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
01

Overview

RG1501 is a gene therapy being developed by RheumaGen in collaboration with the University of Colorado Anschutz Medical Campus for the treatment of multiple sclerosis (MS). The therapy involves editing the Human Leukocyte Antigen (HLA) gene, often referred to as the "immune gene," which is expressed on the surface of most cells and helps the immune system distinguish between self and non-self. By modifying these genes, RG1501 aims to re-educate the immune system to prevent the autoimmune destruction of the myelin sheath in the central nervous system. Currently in the pre-IND (Investigational New Drug) stage, RG1501 is part of a broader pipeline of gene-edited therapies targeting autoimmune diseases, including rheumatoid arthritis and type 1 diabetes.

02

Targets

MHC II (Major histocompatibility complex class II receptor)

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