Technology Bundle ID

Minibody for Conditioning prior to Hematopoietic Stem Cell and Progenitor Cell Transplantation

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Lead Inventors
Andre Larochelle (NHLBI)
Daisuke Araki (NHLBI)
Diogo Magnani (University of Massachusetts Medical School)
Zhirui Wang (University of Colorado Denver)
Development Stages
Patient conditioning is a critical initial step in hematopoietic stem and progenitor cell (HSPC) transplantation procedures to enable marrow engraftment of infused cells. Conditioning regimens have traditionally been achieved by delivering cytotoxic doses of chemotherapeutic agents and radiation. However, these regimens are associated with significant morbidity and mortality, and cannot be used safely in elderly or subjects with comorbidities. Scientists at the National Heart, Lung, and Blood Institute (NHLBI), the University of Massachusetts Medical School (UMMS), and the University of Colorado (CU) have developed a recombinant bivalent (bi) single-chain variable fragment (scFV) minibody targeting the cMPL receptor on HSPCs, fused with diphtheria toxin (DT) truncated at residue 390 (DT390-biscFV(cMPL)) that can be used to deplete HSPCs with increased safety in patients undergoing HSPC transplantation.
Commercial Applications
  • Use as a novel conditioning regimen with increased safety for patients undergoing transplantation
  • Investigational tool to study hematopoietic stem and progenitor cell (HSPC) biology and improve gene and cell therapies
  • Treatment of leukemia
Competitive Advantages
  • Increased safety for patients undergoing transplantation
  • Limits off-target effect
  • Short half-life allowing rapid clearance before cell re-infusion
  • Better penetrates into deep tissue, such as bone marrow niche
  • More efficiently dimerizes cMPL receptor than the whole IgG

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