Scientific Approach

At Jasper, we are dedicated to bringing cures for patients suffering from mast and hematopoietic stem cell diseases.

In mast cell diseases our approach is to directly target and deplete the cells that are responsible for the inflammatory response in skin and lungs.

In Low to Intermediate Risk MDS our approach is to target the diseased stem cells in the bone marrow that interfere with the normal production of blood and immune cells.

Our approach to improve curative hematopoietic stem cell transplants involves: 1) using a very targeted approach to develop a safer, more effective alternative to toxic conditioning regimens that could expand the use of curative stem cell transplants and gene therapies; and 2) advancing a novel platform to engineer hematopoietic stem cells that address the limitations of transplant grafts and increase curative potential.

Addressing Limitations Of Transplant Conditioning

Hematopoietic stem cell transplantation is among the most widely practiced forms of cellular therapy and can cure many diseases. It has the potential to cure an even greater number of diseases but toxicity from current chemotherapy conditioning technologies limits its broad application and the number of patients able to benefit. Currently, patients must receive non-specific conditioning agents to prepare their bone marrow for hematopoietic stem cell transplantation.

The standard of care is chemotherapy/radiation, which has several limitations:

  • Major toxicities & adverse events (cancer, infection, infertility)
  • Hospitalization in isolation
  • Life-threatening risk

Our highly differentiated anti c-Kit antibody briquilimab, is designed to address these challenges.

Addressing Limitations Of Transplant Grafts

Allogeneic or autologous gene edited hematopoietic stem cell therapy have the potential to provide curative benefit to patients with hematologic cancers, genetic diseases (e.g., beta thalassemia and sickle cell disease), severe or refractory autoimmune diseases, and solid organ transplant.

However, only a minority of eligible patients receive a transplant because of limitations of transplant grafts, including:

  • Clinical relapse
  • Failed or poor engraftment
  • Graft vs. host disease (GvHD)
  • Long-term immunosuppression

We are working to address these challenges with our engineered hematopoietic stem cells (mRNA stem cell graft).

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