Ex vivo-expanded highly pure ABCB5

ABCB5 GMP manufacturing advanced therapy medicinal product chronic wound mesenchymal stromal cells venous ulcer

Journal

Cytotherapy
ISSN: 1477-2566
Titre abrégé: Cytotherapy
Pays: England
ID NLM: 100895309

Informations de publication

Date de publication:
02 2021
Historique:
received: 14 05 2020
revised: 05 08 2020
accepted: 29 08 2020
pubmed: 5 10 2020
medline: 7 10 2021
entrez: 4 10 2020
Statut: ppublish

Résumé

Mesenchymal stromal cells (MSCs) hold promise for the treatment of tissue damage and injury. However, MSCs comprise multiple subpopulations with diverse properties, which could explain inconsistent therapeutic outcomes seen among therapeutic attempts. Recently, the adenosine triphosphate-binding cassette transporter ABCB5 has been shown to identify a novel dermal immunomodulatory MSC subpopulation. The authors have established a validated Good Manufacturing Practice (GMP)-compliant expansion and manufacturing process by which ABCB5 As of now, 12 wounds in nine patients have been treated with 5 × 10 The authors describe here their GMP- and European Pharmacopoeia-compliant production and quality control process, report on a pre-clinical dose selection study and present the first in-human results. Together, these data substantiate the idea that ABCB5

Sections du résumé

BACKGROUND AIM
Mesenchymal stromal cells (MSCs) hold promise for the treatment of tissue damage and injury. However, MSCs comprise multiple subpopulations with diverse properties, which could explain inconsistent therapeutic outcomes seen among therapeutic attempts. Recently, the adenosine triphosphate-binding cassette transporter ABCB5 has been shown to identify a novel dermal immunomodulatory MSC subpopulation.
METHODS
The authors have established a validated Good Manufacturing Practice (GMP)-compliant expansion and manufacturing process by which ABCB5
RESULTS
As of now, 12 wounds in nine patients have been treated with 5 × 10
CONCLUSIONS
The authors describe here their GMP- and European Pharmacopoeia-compliant production and quality control process, report on a pre-clinical dose selection study and present the first in-human results. Together, these data substantiate the idea that ABCB5

Identifiants

pubmed: 33011075
pii: S1465-3249(20)30851-3
doi: 10.1016/j.jcyt.2020.08.012
pmc: PMC8310651
mid: NIHMS1714686
pii:
doi:

Substances chimiques

ABCB5 protein, human 0
ATP Binding Cassette Transporter, Subfamily B 0

Banques de données

ClinicalTrials.gov
['NCT03257098', 'NCT03267784', 'NCT03529877', 'NCT03860155']

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

165-175

Subventions

Organisme : BLRD VA
ID : I01 BX000516
Pays : United States
Organisme : RRD VA
ID : I01 RX000989
Pays : United States
Organisme : NEI NIH HHS
ID : R01 EY025794
Pays : United States
Organisme : NEI NIH HHS
ID : R24 EY028767
Pays : United States

Informations de copyright

Copyright © 2020 International Society for Cell & Gene Therapy. Published by Elsevier Inc. All rights reserved.

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Auteurs

Andreas Kerstan (A)

Department of Dermatology, Venereology, and Allergology, University Hospital Würzburg, Würzburg, Germany.

Elke Niebergall-Roth (E)

TICEBA GmbH, Heidelberg, Germany.

Jasmina Esterlechner (J)

TICEBA GmbH, Heidelberg, Germany.

Hannes M Schröder (HM)

TICEBA GmbH, Heidelberg, Germany.

Martin Gasser (M)

Department of Surgery, University Hospital Würzburg, Würzburg, Germany.

Ana M Waaga-Gasser (AM)

Department of Surgery, University Hospital Würzburg, Würzburg, Germany; Renal Division, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Matthias Goebeler (M)

Department of Dermatology, Venereology, and Allergology, University Hospital Würzburg, Würzburg, Germany.

Katrin Rak (K)

Department of Dermatology, Venereology, and Allergology, University Hospital Würzburg, Würzburg, Germany.

Philipp Schrüfer (P)

Department of Dermatology, Venereology, and Allergology, University Hospital Würzburg, Würzburg, Germany.

Sabrina Endres (S)

Department of Dermatology, Venereology, and Allergology, University Hospital Würzburg, Würzburg, Germany.

Petra Hagenbusch (P)

Department of Dermatology, Venereology, and Allergology, University Hospital Würzburg, Würzburg, Germany.

Korinna Kraft (K)

RHEACELL GmbH & Co. KG, Heidelberg, Germany.

Kathrin Dieter (K)

RHEACELL GmbH & Co. KG, Heidelberg, Germany.

Seda Ballikaya (S)

TICEBA GmbH, Heidelberg, Germany.

Nicole Stemler (N)

TICEBA GmbH, Heidelberg, Germany.

Samar Sadeghi (S)

TICEBA GmbH, Heidelberg, Germany.

Nils Tappenbeck (N)

RHEACELL GmbH & Co. KG, Heidelberg, Germany.

George F Murphy (GF)

Department of Dermatology, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Dennis P Orgill (DP)

Division of Plastic Surgery, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts, USA.

Natasha Y Frank (NY)

Department of Medicine, VA Boston Healthcare System, Boston, Massachusetts, USA; Division of Genetics, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts, USA; Transplant Research Program, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, Massachusetts, USA.

Christoph Ganss (C)

TICEBA GmbH, Heidelberg, Germany; RHEACELL GmbH & Co. KG, Heidelberg, Germany.

Karin Scharffetter-Kochanek (K)

Department of Dermatology and Allergic Diseases, University Hospital, Ulm, Germany.

Markus H Frank (MH)

Department of Dermatology, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts, USA; Transplant Research Program, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA; Harvard Stem Cell Institute, Harvard University, Cambridge, Massachusetts, USA; School of Medical and Health Sciences, Edith Cowan University, Perth, Australia.

Mark A Kluth (MA)

TICEBA GmbH, Heidelberg, Germany; RHEACELL GmbH & Co. KG, Heidelberg, Germany. Electronic address: andreas.kluth@ticeba.com.

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