NOVEL METHOD OF GENERATING HEMATOPOIETIC STEM CELLS

Tech ID: 25496 / UC Case 2015-230-0

Invention Novelty

This invention may enable the re-engineering of mature human endothelium (blood vessels) into blood-producing hematopoietic stem cells (HSCs).

Value Proposition

The current roadblocks to HSC therapies include the rarity of matched donors for bone marrow transplant, engraftment failures, common shortages of donated blood, and the inability to expand HSCs ex vivo in large numbers. These major obstacles would cease to exist if an extensive, bankable, inexhaustible, and patient-matched supply of blood were available. The recent validation of hemogenic endothelium (blood vessel cells lining the vessel wall that give rise to blood stem cells) has introduced new possibilities in HSC therapy.  Thus, the inventors aimed to understand the requirements for the process of endothelial to hematopoietic transition and to re-engineer mature human endothelium into blood-producing blood HSCs.

Engineering functional HSCs from mature endothelium may present the following advantages: 

  • Enable large-scale production for clinical use
  • Donors could potentially be matched, leading to increased engraftment and decreased rejection of bone marrow transplants
  • Cell renewal properties could be capitalized on while minimizing malignancy risk
  • Potential for multi-lineage long term repopulating capacity, unlike HSCs derived from embryonic or induced pluripotent stem cells
  • Longer shelf life compared to cord blood
  • Requires only one donor compared to cord blood which requires two donors for adults needing bone marrow transplantation, which increases the exposure to antigens

Technology Description

Scientists at the University of California, San Francisco have developed a system for reprogramming endothelial cells into blood like cells (being evaluated currently for bona fide blood-producing HSC activity) using episomal non-integrating vectors.  They envision long-term banking of endothelial cells (which can be kept at -80°C conditions), indexed with HLA subtypes and other phenotype analysis important for transplantation, comprising a large database. When a bone marrow transplant is needed and matched against the endothelial database, then the endothelial cell line is thawed and reprogrammed into HSCs for transplantation using this novel approach.  

Application

Blood transplantation

Looking for Partners

To develop and commercialize this technology as an alternative source of patient-matched blood

Stage of Development

Preclinical

Related Materials

Data Availability

Under NDA/CDA

Patent Status

Country Type Number Dated Case
Patent Cooperation Treaty Reference for National Filings WO2016201133 12/15/2016 2015-230
 

Patent Pending

Contact

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Other Information

Keywords

Hematopoietic Stem Cells, Episomal Reprogramming, Endothelial Cells, Bone Marrow Transplant, Blood Banking

Categorized As