We have developed our proprietary RED PLATFORM® to generate red blood cells and genetically engineer them into a new class of cellular medicines, called Red Cell TherapeuticsTM (RCTs). Our RED PLATFORM harnesses both the unique properties of red blood cells and the creative potential of genetic engineering to deliver on the promise of cellular therapy and transform how diseases are treated.
Red Cell Therapeutics are engineered to express biotherapeutic proteins within or on their cell surface to create highly selective, potent and allogeneic cellular medicines aimed at treating a range of diseases. They can potentially be used to replace missing enzymes for patients living with certain rare enzyme deficiencies, kill tumors in cancer and regulate the immune system for the treatment of autoimmune diseases.
THERAPEUTIC MODALITIES
RCTTM Product Candidates can be engineered in three distinct ways to treat disease
By leveraging three distinct therapeutic modalities—cellular shielding, immune system activation and tolerance induction—our initial RCTs have the potential to improve the treatment of rare enzyme deficiencies, cancer and autoimmune diseases.
Rare Diseases
Enzyme Replacement
Cellular Shielding andEnzyme Deficiencies
RCTs are engineered to express therapeutic enzymes within the cell and to shield those enzymes from the immune system.
Cellular Shielding andEnzyme Deficiencies
RCTs are engineered to express therapeutic enzymes within the cell and to shield those enzymes from the immune system. As RCTs circulate, they are designed to degrade the toxic metabolites that accumulate in the blood stream. Compared to current therapeutic approaches, RCT™ product candidates may have a more sustained treatment effect given the 120-day circulating time of red blood cells and may have a lower incidence of immune-driven adverse events, including the formation of neutralizing antibodies to the therapeutic enzyme, which results in a reduction in efficacy.
Cancer
Immune Modulation
Immune System Activation in Cancer
RCTs can be engineered to co-express combinations of co-stimulatory molecules...
Immune System Activation in Cancer
RCTs can be engineered to co-express combinations of co-stimulatory molecules on the cell surface to directly engage T and NK cells to activate the adaptive and innate immune systems. The goal is to stimulate these cells to proliferate, activate, migrate and, ultimately, to attack and kill tumors. This approach has the potential to improve anti-tumor activity and overcome resistance to immunotherapy.
Additionally, we are advancing RCT product candidates that function as artificial antigen presenting cells, or RTX-aAPCs. Our initial focus is on expressing tumor antigens fused to major histocompatibility complex class I, or MHC I, on the red cell surface. We expect to induce highly selective tumor killing by stimulating the immune system to target tumors in an antigen-specific manner.
Autoimmune Diseases
Antigen Presentation
Tolerance Induction inAutoimmune Diseases
We have also engineered RCTs to express disease-causing antigens on their surface.
Tolerance Induction inAutoimmune Diseases
We have also engineered RCTs to express disease-causing antigens on their surface. When these RCTs are processed by the reticuloendothelial system, the antigens are expected to be re-presented to the immune system. This approach is intended to induce tolerance by retraining the immune system to no longer respond to self-antigens as foreign. Restoration of immune tolerance could lead to cures in certain autoimmune diseases.
LessSEE OUR RED CELL THERAPEUTICS IN ACTION
MANUFACTURING OUR RED CELL THERAPEUTICS
With the RED PLATFORM®, Rubius Therapeutics can generate a wide variety of allogeneic, ready-to-use RCTTM product candidates with our proprietary process:
- CD34+ hematopoietic precursor cells are collected by apheresis from a healthy O negative donor and purified
- These precursor cell populations are genetically engineered with a lentiviral vector, or gene cassette, to express one or more biotherapeutic proteins
- The cells are then exposed to media in a bioreactor to promote further expansion and differentiation until the nucleus is ejected, resulting in a mature reticulocyte
- Based on our manufacturing release criteria, the remaining cell, or RCT, has the same characteristics as a normal red blood cell and now has the biotherapeutic protein(s) of interest inside or on the cell surface to treat the intended disease
- A donation from one O negative donor can result in hundreds to thousands of doses, depending on the intended therapeutic application
- We can generate a broad pipeline of RCT product candidates by changing only the gene cassettes that encode the biotherapeutic protein(s)
