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UM171 Molecule
ExCellThera’s most advanced technology is based around the UM171 small molecule. UM171 is a first in class activator of a novel protein leading to the degradation of a repressor complex and the activation genes involved in the expression of stem and immune cells.
X Y UM171 UM171

Stem Cells

  • Fast & persistent engraftment
  • Faster immune reconstitution

Mast Cells

  • Less gut GvHD
  • Reduced TRM

Dendritic Cells

  • Anti-leukemic effect
  • Epithelium (gut) integrity

Cell Expansion

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Unique and Differentiated Graft

ECT-001 offers an increased dose of stem cells for fast engraftment, and provides a high dose of tolerogenic
mast cells, dendritic mature cells and progenitors.

The UM171 molecule permits for the creation of a new
graft, made up of approximately 50% immune cells, contributing to various positive clinical outcomes, such as a reduction of gut Graft versus Host Disease (GvHD).

CONTROL ECT-001 7 days Dendritic Cells: reduces gut damage HSPC: fast engraftment Mast Cells: reduces gut GvHD tSNE-2 tSNE-1
CONTROL ECT-001 7 days tSNE-2 tSNE-1 Dendritic Cells: reduces gut damage Stem Cells (HSPC) fast engraftment Mast Cells: reduces gut GvHD
>25x 10 7 10 6 10 5 10 4 10 3 10 2 10 1 Cell dose (/kg) CD34+ HSPC HSC Dendritic Mast >18x >1,000x >600x >8000x
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Drug Product
The ECT-001-CB drug product is safe and manufactured in 7 days in small culture volumes.




CD34 - Cells CD34 + Cells UM171 Wash Cell Expansion Cell Selection Cell INFUSIOn Unexpanded Cryopreserved T Cells Expanded Cryopreserved Stem Cells
Cell infusion CD34 + Cells Cell Selection Expanded Cryopreserved Stem Cells (HSPC) UM171 Cell Expansion Wash Unexpanded Cryopreserved T Cells CD34 - Cells Cell Selection