Autoimmune

After years of treating autoimmune diseases with chronic doses of small molecules and antibodies, we're entering a new paradigm with potential one-time therapies that could produce long-term remission. Using gene editing, we aim to engineer immune cells that correct the root cause of many autoimmune diseases in a robust and specific way with the hope of avoiding many toxicities of traditional treatments.

The immune system is the body’s defense against infection and disease. Autoimmune diseases occur when immune cells confuse “non-self” (pathogens like viruses and bacteria) with “self” (healthy organs and tissue). In many autoimmune diseases, B-cells play a critical role in starting or sustaining this self-directed immune attack. Encouraging data have shown the promise of using patient’s own (autologous) T-cells to make chimeric antigen receptor (CAR) T cells that eliminate unhealthy B-cells and reset the immune system.

We believe that CRISPR-based gene editing will drive the next generation of therapies for autoimmune diseases. Using CRISPR, we can manufacture CAR T cell therapies from healthy donors (allogeneic) that offer several potential advantages:

• Immediate availability and broader access: administered off-the-shelf with no need for patient apheresis or bespoke manufacturing
• Greater consistency and manufacturability: each batch yields many doses using starting material derived from healthy donors
• Flexible dosing: ability to re-dose
• Precision therapy: targeted gene insertion using CRISPR technology