This technology uses lung progenitor cells generated from human stem cell-derived lung organoids in vitro as therapeutics for various pulmonary diseases.
Unmet Need: Cell replacement therapy substrate to treat lung disease
Lung transplantation, currently the standard of care for end-stage lung disease, is limited by a shortage of donor organs and immunological challenges. Gene therapy offers an alternative treatment option to target defective genes in lung tissue, but permanent replacement of epithelial compartments of the distal lung may be a more reliable approach to ensure precise gene expression. Cell replacement therapy offers a promising direction for both prevention and treatment of terminal lung disease. For this type of therapy, human pluripotent stem cells (hPSCs) or induced pluripotent stem cells (iPSCs) may be promising candidates to recover the distal lung following injury.
The Technology: Cell replacement therapy using stem cell-derived, developmentally mature epithelial progenitors
This technology describes a technique for differentiating human pluripotent stem cells (hPSCs) or induced pluripotent stem cells (iPSCs) into distal lung progenitor cells in the form of lung organoids that have transcriptomic signatures resembling either basal or secretory cells. These cells are presented as a candidate substrate for cell therapies to benefit patients suffering from severe, terminal lung disease. The use of stem cells enables patient-specific expansion of cells that are less likely to be rejected upon introduction and engraftment in vivo.
This technology has been validated in vivo in a rat model.
Applications:
- Personalized cell therapy approach for lung disease
- Expansion of induced pluripotent stem cells or human embryonic stem cells into basal cells and secretory cells
- Generation of mature endothelial cell organoids
- Administering human subject-specific lung cells from organoids generated in vitro for patient-specific therapy
- Treatment of childhood interstitial lung disease (chILD), idiopathic pulmonary fibrosis (IPF), chronic obstructive pulmonary disease (COPD), familial idiopathic pulmonary fibrosis, neonatal respiratory distress, an epithelial lung disease, a disease initiated by chronic epithelial injury, or an interstitial lung disease
Advantages:
- Patient-specific, personalized medicine intervention
- Lower probability of host rejection with the use of patient-specific stem cells
- Opportunities for both preventative and therapeutic interventions
- High expansion potential in vitro offers the opportunity for high yield
- Overcome shortages in donor organs for lung transplantation procedures
Lead Inventor:
Hans-Willem Snoeck, Ph.D.
Patent Information:
Patent Pending(WO/2024/233940)
Related Publications:
Leko IM, Schrode N, Torres J, Pezet M, Thimraj TA, Beaumont KG, Snoeck HW. “Generation and expansion of transitional lung organoids from human pluripotent stem cells.” bioxRiv. 2023 Feb 1.
Matkovic Leko I, Schneider RT, Thimraj TA, Schrode N, Beitler D, Liu HY, Beaumont K, Chen YW, Snoeck HW. “A distal lung organoid model to study interstitial lung disease, viral infection and human lung development.” Nat Protoc. 2023 May 10; 18(7): 2283-2312.
Rodrigues Toste de Carvalho AL, Liu HY, Chen YW, Porotto M, Moscona A, Snoeck HW. “The in vitro multilineage differentiation and maturation of lung and airway cells from human pluripotent stem cell–derived lung progenitors in 3D.” Nat Protoc. 2021 Mar 1; 16(4): 1802-1829.
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