Recent treatment advances have focused on activating the immune system against cancer. Despite this progress, existing immunotherapies are effective in a relatively small number of patients and types of cancer. Furthermore, even when therapies are initially effective, the disease often eventually progresses and no longer responds to treatment.
RCTTM product candidates can be engineered to express combinations of co-stimulatory molecules and cytokines on their cell surface to directly engage both the adaptive immune system and the innate immune system. By stimulating both powerful arms of the immune system, RCTs may be better able to destroy cancer cells to improve anti-tumor activity and overcome resistance to immunotherapy.
We believe RCTs will have broad therapeutic application across a range of solid tumors.
Autoimmune diseases are the result of a hyperactive immune response that mistakes healthy tissue as foreign. Over time, these attacks on healthy cells lead to disease. Current therapies are generally administered on a lifelong basis, initially work for only a subset of patients, tend to lose effectiveness over time and are often associated with serious side effects, including opportunistic infections, lymphoma and, in some cases, severe or fatal reactions.
While the triggers of most autoimmune diseases remain unknown, it is generally understood that disease is the result of a loss of tolerance to one’s own cells. The accepted model of disease assumes an inherent genetic susceptibility followed by an environmental trigger, which leads to a breakdown of immune regulation. In principle, restoration of peripheral tolerance should provide patients with a partial response or a complete cure.
RCTs can be engineered to express specific autoimmune disease-associated antigens either within the cell or on the cell surface to take advantage of how the body normally maintains self-tolerance, thereby retraining the immune system to no longer see self-antigens as foreign. In addition, RCTs can be engineered to express immune modulating cytokines, enzymes or inhibitory signals, which may have the potential to enhance the tolerogenic effects of the RCTs. The resulting restoration or induction of immune tolerance (also known as tolerance induction) may lead to more effective treatments, and, in some cases — potentially even cures — for certain autoimmune diseases, such as Type I diabetes, celiac disease and multiple sclerosis.
Rubius Therapeutics is also exploring how to generate T cell-directed immune tolerance as well as arming RCTs with other immunomodulators and targeting moieties to induce tolerance. Using this approach, RCTs may be able to be engineered to prevent immunogenicity to enzyme replacement therapy and gene therapy.
At Rubius Therapeutics, we are focused on developing an entirely new class of medicine to treat cancer and autoimmune diseases. We are conducting several clinical trials to demonstrate the safety and efficacy of these potential treatments. These investigational treatments have not been approved by regulatory authorities, such as the United States Food and Drug Administration (FDA).
Patient access programs allow patients with immediately life-threatening conditions or serious diseases to gain access to an investigational therapy outside of clinical trials. As we carefully consider the health and well-being of patients and families, our first commitment is to avoid inadvertently doing harm while the safety of our treatments is still being evaluated.
At this time, we are not able to provide access to our investigational therapies outside of a clinical trial setting. We believe the best way to develop safe and effective treatments that have a positive benefit for the largest number of patients is to continue our rigorous clinical development program.
We encourage any person interested in learning more about participating in one of our clinical trials to discuss their eligibility with their physician or to visit www.rubiustrials.com.