RheumaGen is pioneering a novel approach to treating autoimmune diseases through its innovative HLA gene-editing technology. Human Leukocyte Antigens (HLA) are critical genetic markers that help the immune system distinguish between the body’s own cells and foreign invaders. In individuals with autoimmune diseases, these HLA markers mistakenly identify healthy cells as threats, leading to an inappropriate immune response. Unlike traditional therapies that focus on alleviating symptoms or suppressing the immune system, RheumaGen's technology directly targets the HLA gene itself. By employing advanced gene editing techniques, the company aims to modify the HLA gene in a way that prevents the immune system from attacking healthy cells, thereby addressing the root cause of autoimmune disorders.

The company's lead preclinical therapy, RG0401, exemplifies this approach by editing the HLA gene to create molecules that mimic those found in individuals resistant to rheumatoid arthritis (RA). This precision editing allows the immune system to remain intact and functional, enabling it to combat genuine threats like infections while halting the autoimmune attack on the body’s own collagen cells. RheumaGen's technology not only promises a one-time outpatient procedure for patients with refractory RA but also holds potential for treating a range of other autoimmune conditions, including ankylosing spondylitis, multiple sclerosis, type 1 diabetes, and celiac disease. This groundbreaking method represents a significant shift in the treatment paradigm for autoimmune diseases, focusing on genetic modification to restore immune balance without compromising overall health.

United States

RheumaGen is the only cell and gene therapy company editing the HLA gene to cure autoimmune diseases at their source.

RheumaGen

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