Cell Expansion

UM171

UM171 is the Standard for Ex Vivo HSC Expansion in Cell & Gene Therapy.

UM171 discovery and optimization published in:

Full results of initial clinical trial published in:

UM171 mechanism of action published in:

Best molecule for HSC expansion¹
First-in-class mechanism of action
Clinically validated in over 100 patients²
Used under license by bio-pharmas and academia
Pre-formulated GMP-grade single-use vials
Ready for integration into cell & gene therapies
Supported by an FDA Drug Master File (DMF)

(1)Science 2014; Lancet Hematol 2020; Cell Stem Cell 2021; Mol Ther Methods Clin Dev 2018 and many others; Collaboration in iPSCs with Astellas; Collaborations with several cell & gene therapy companies.

(2)100+ patients treated with UM171-expanded cells; initial clinical trial results published by Cohen et al. in Lancet Haematology (2020).

Cell Expansion

Cell expansion

& Rejuvenation

Cell Expansion

Protection Against

Negative Impacts of Culture

Ex vivo culture has negative impacts on cells

UM171 counteracts HSC exhaustion and aging

	Youthful Cells (uncultured)
Intracellular ROS levels	Low
Protein synthesis	Low
Reconstitution activity	High
ATP production	Low
Lysosomal activity	High
Fatty and amino acid oxidation capacity	High

	Aged Cells / Cells Cultured without UM171
Intracellular ROS levels	High
Protein synthesis	High
Reconstitution activity	Low
ATP production	High
Lysosomal activity	Low
Fatty and amino acid oxidation capacity	Low

	Cells Cultured with UM171
Intracellular ROS levels	Low
Protein synthesis	Med
Reconstitution activity	High
ATP production	Low
Lysosomal activity	High
Fatty and amino acid oxidation capacity	High

(modified from Nakamura-Ishizu A., Dev Cell. 2020)

Youthful HSC

Without UM171

Cell Culture / Manipulation

With UM171

Multiple Youthful HSCs

Exhausted HSC

Cell Expansion

UM171

Specifications

Active Compound Name	UM0128171
Activity	Enhances human hematopoietic stem cell self-renewal in vitro
Active Compound Structure	(1r,4r)-N1 -(2-benzyl-7-(2-methyl-2H-tetrazol- 5-yl)-9H-pyrimido[4,5-b]indol-4- yl)cyclohexane-1,4-diamine dihydrobromide dihydrate
Active Compound Molecular Weight	453.55 g/mol (free base)
Active Compound CAS Number	1448724-09-1
Drug Master File (DMF) Number	MF028635 (single use vials)
Alternative Name	UM171
Intended Use	Intended for ex vivo use only
Quality	Product is manufactured, packaged and tested (quality controlled) in compliance with applicable Good Manufacturing Practices (GMP) 21CFR 210/211 requirements and specifications
Purity	> 95%
Endotoxin Level	< 10 EU/mL
Transport Information	Not a hazardous substance or mixture according to the Globally Harmonized System (GHS) Not regulated under DOT, TDG, MEX, ICAO (air), IATA, IMDG, RID, ADR or ADN