Haematopoietic stem / progenitor cells and methods for their production

A culture system using defined growth factors and signaling agents differentiates iPSCs into CD34+ hematopoietic stem/progenitor cells with multilineage engrafting capacity, addressing the challenges of existing differentiation methods and donor-host mismatch.

WO2025199576A1 Publication Date: 2025-10-02MURDOCH CHILDRENS RES INST
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Patent Information

Application Number
PCT/AU2025/050292
Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
Priority Date
2024-03-25
Filing Date
2025-03-25
Publication Date
2025-10-02

AI Technical Summary

Technical Problem

Existing methods struggle to differentiate pluripotent stem cells into transplantable hematopoietic stem/progenitor cells with multilineage engrafting capacity, particularly due to difficulties in distinguishing between cells with and without repopulating activity, and the challenge of donor-host mismatch in allogenic transplants.

Method used

A fully defined culture system using specific growth factors and signaling agents, including WNT agonists, ACTIVIN antagonists, and retinoic acid, to guide iPSCs through a HOXA-positive differentiation pathway mimicking AGM-like hematopoiesis, resulting in CD34+ hematopoietic stem/progenitor cells capable of cryopreservation and intravenous transplantation.

Benefits of technology

The method produces hematopoietic stem/progenitor cells with multilineage engrafting capacity and repopulating activity, similar to human umbilical cord blood-derived cells, suitable for therapeutic applications.

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Abstract

The invention relates to differentiating a pluripotent stem cell (PSC) into a definitive haematopoietic stem / progenitor cell which is capable of multilineage engraftment, and to therapeutic uses of such haematopoietic stem / progenitor cells.
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