Autoimmune diseases such as lupus, type 1 diabetes, inflammatory bowel disease (Crohn’s disease or ulcerative colitis) and rheumatoid arthritis are complex conditions involving multi-factorial mechanisms of action and patient heterogeneity. Treating them with CGT requires a comprehensive understanding of the specific disease biology and a reliable source of cellular starting materials.

CGT Challenges

As researchers look beyond using cell and gene therapy (CGT) for oncology indications and start to focus on autoimmune disorders, they need to adjust their approach. Autoimmune
diseases are highly individualised, and patients have different pathophysiology. Therefore, researchers need to develop personalised therapies that are tailored to the specific immune
profiles and needs of each patient. They can be autologous (using the patient’s own cells) or allogeneic (using cells from a donor).

A deep understanding of the underlying mechanisms of action is essential. Researchers need to identify specific targets within the immune pathways that can be modulated effectively to restore balance without compromising overall immune function.

Delivery of the CGT must also be optimised for autoimmune disorders. This may involve the use of advanced delivery systems, such as nanoparticles or viral vectors, which can enhance targeting and uptake of the therapies in specific tissues or organs affected by the disease.