RheumaGen

RheumaGen is developing a novel gene-editing therapy to cure rheumatoid arthritis (RA). RheumaGen’s technology alters the Human Leukocyte Antigen (HLA) gene so that the molecule no longer triggers T cells to attack the body’s own healthy collagen – without diminishing the body’s overall immune response. The research team has already successfully modified the HLA molecule and demonstrated RA resistance in mice with humanized immune systems. RheumaGen is preparing an IND submission to the FDA with plans to begin human clinical trials within 18 months. The RA market consists of approximately 5 million patients, 1.7 million of whom have moderate to severe cases. RheumaGen’s initial target market is the approximately 500,000 RA patients whose RA does not respond to currently available treatments. The RheumaGen HLA editing technology also shows promise for the future treatment of ankylosing spondylitis, multiple sclerosis, type 1 diabetes, and celiac disease.