Science
What is HLA?
Human leukocyte antigen (HLA), or “immune gene,” is the fundamental signal of the human adaptive immune system that alerts one’s immune cells to protect the body against antigens (e.g., the flu).
What Causes Autoimmune Disease?
In autoimmune disease, one of your HLA alleles mistakenly identifies a self-protein as foreign, which activates your T cells that cause chronic autoimmune responses.
RheumaGen’s Transformative Therapies
RheumaGen is developing a breakthrough class of cell and gene therapies designed to cure autoimmune diseases at their source. The company is focused on editing the human leukocyte antigen (HLA), or “immune gene,” so that the immune system does not attack healthy cells.
With a precision edit to a DNA marker(s) of the HLA gene, RheumaGen’s cell and gene therapies are designed only to make a patient’s HLA molecules mirror those of a person resistant to a particular autoimmune disease, while maintaining the rest of the immune system as normal. These therapies change harmful HLA alleles to healthy ones and prevent T cells from activating chronic autoimmune responses, thereby halting not only cell and tissue degradation but also the entire disease process with this approach.
How it Works
Here is the basic clinical process of how our therapies work:
- First, we collect the hematopoietic stems cells (HSCs) found in the patient’s blood.
- Second, we introduce our engineered HLA allele using a gene-editing platform.
- Next, we transfer the precisely edited HSCs back to the treatment center for a one-time infusion of the patient.
- Finally, within days, those edited HSCs will begin producing new antigen presenting cells with our precisely modified HLA molecules that no longer activate T cells and prevent chronic autoimmune responses.
The Power of HLA Gene Editing
Powered by its CGT technology and decades of immunology, histocompatibility, and clinical expertise, RheumaGen has demonstrated strong preclinical data indicating that HLA gene-editing to treat autoimmunity is not only safe but also holds great promise to cure autoimmune diseases.