Rubius Erythrocyte Design (RED)™ Platform

Rubius Therapeutics is using breakthrough science to develop an entirely new class of drugs with the potential to treat many areas of human disease. The Rubius Erythrocyte Design™ (RED) platform enables Rubius scientists to genetically modify hematopoietic stem cells and to turn them into red blood cells in culture. By modifying the genes of the hematopoietic progenitors, Rubius creates Red-Cell Therapeutics™ (RCTs): functionalized human red blood cells that express biotherapeutic proteins, either on their surface or inside the cell. These mature, functionalized red blood cells have great therapeutic potency for a wide array of diseases.

As a therapeutic drug class, RCTs are highly advantaged by virtue of their unique ability to circulate in the body, engage and modulate the immune system, and elicit profound effects on human biology. Rubius has the capability to design RCTs with a variety of metabolic and functional potencies. Because all red blood cells eject their nuclei, the final gene-free Red-Cell Therapeutics are safer and more easily controlled than gene and cell therapies. With more than 200 prototypes generated to date, the company has demonstrated the powerful curative properties of its modified enucleated cells in preclinical models, and has parallel drug discovery and development programs underway.

Red-Cell Therapeutics are being developed to address unmet medical needs in the areas of oncology, autoimmune disease, rare disease, cardiovascular and metabolic disease and infectious disease. For example, Rubius has created RCTs expressing multiple surface proteins capable of inhibiting cancer cell growth and activating the patients' immune system to clear cancer cells. This approach can be further enhanced by expressing tumor antigen binding proteins on the surface of RCTs, resulting in enhanced potency and selectivity. Alternatively, RCTs have been engineered to express enzymes for replacement therapy in patients living with a variety of rare diseases – examples of these include RCTs that can continually remove unwanted metabolic products from the bloodstream. RCTs also have powerful immunomodulatory effects - they can be engineered with protein or peptide antigens to induce tolerance to those antigens – a capability that holds promise to treat severe autoimmune diseases.