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, culture and mature hematopoietic stem cells to create allogeneic, functionalized Red-Cell Therapeutics™.
These “off-the-shelf” biotherapeutics display receptor agonists, antigens and binders, enzymes, and combinations thereof on the cell surface or in the cytosol.
Red cells have unique properties that provide researchers with significant drug development advantages:
- Enucleation post-genetic engineering ensures that RCTs lack the cellular machinery to expand or transform
- Immuno-privileged: Specific surface proteins shield against immunological detection
- Sequestered in the vasculature: The ability to target specific cells, tumors, and tissues with limited off-target effects
- Circulate for 3-4 months: Durable activity and the ability to optimize dose
Because all human red cells eject their nuclei, the final gene-free Red-Cell Therapeutics are safer and more easily controlled than gene and cell therapies.
- The use of O(-) universal donor blood as a source of material eliminates the risk of major blood type mismatch, allowing us to use a single donor to generate broadly applicable, off-the-shelf products
- We engineer hematopoietic precursor cells to express millions of copies of therapeutic moieties per cell. The Rubius Erythrocyte Design PlatformTM enables high throughput early lead development in 4-6 weeks
- RCTs™will be manufactured using a GMP-compliant production model that leverages commonly available, validated, scalable bioreactors in a single, consistent CMC process
- We deliver allogeneic products that can be administered to the patient in less than 30 minutes
Rubius Therapeutics is developing Red-Cell Therapeutics (RCTs) as a new class of medicines to address a wide array of indications, with leading applications in cancer, rare and autoimmune disease, as well as additional potential in hemophilia, infectious and metabolic diseases.
Our lead programs are targeting diseases with no acceptable treatment options. Immuno-privileged presentation of our therapeutics increases efficacy, while the long half-life improves compliance and convenience. Clinical trials are generally short and biomarkers can be used as a surrogate for efficacy and approval.
We can target, starve, penetrate, and destroy tumors. We have demonstrated checkpoint immunomodulation and have been able to show that co-expressing two different moieties on a single cell generates a synergistic response. We are building a combinatorial portfolio of binding, stimulation, and killing modalities in a range of hematological and solid tumors.
AUTOIMMUNE DISEASE AND BEYOND
RCTs™ enable precise targeting of specific components of the immune system. We are leveraging our B-cell and T-cell work in oncology to build similar and mirror image constructs in autoimmune diseases. RCTs™ have been shown to induce dramatic antigen-specific tolerance, and thus, functional cure in a range of diseases. The in vivo functional rescue and survival data generated to date in a Multiple Sclerosis model is unprecedented.