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The aim of this clinical trial is to investigate the safety and efficacy of allo-APZ2-OTS administered intravenously to subjects with recessive dystrophic epidermolysis bullosa (RDEB) compared to placebo. An additional baseline-controlled open-label arm will be included to investigate the safety and efficacy of allo-APZ2-OTS administered intravenously to subjects with JEB and to RDEB subjects < 1 year.

Start Date01 Feb 2023

Sponsor / Collaborator

RHEACELL GmbH & Co. KG

100 Clinical Results associated with RHEACELL GmbH & Co. KG

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0 Patents (Medical) associated with RHEACELL GmbH & Co. KG

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28

Literatures (Medical) associated with RHEACELL GmbH & Co. KG

01 Sep 2023·Cytotherapy

Corrigendum to ABCB5+ mesenchymal stromal cells facilitate complete and durable wound closure in recessive dystrophic epidermolysis bullosa [Cytotherapy 25 (2023) 782–788/1562]

Author: Dieter, Kathrin ; Tolar, Jakub ; Niebergall-Roth, Elke ; Kluth, Mark A ; Frank, Natasha Y ; Fluhr, Silvia ; McGrath, John A ; Frank, Markus H ; Kiritsi, Dimitra ; Ganss, Christoph ; Daniele, Cristina

05 Jul 2023·Cells

Drug Regulatory-Compliant Validation of a qPCR Assay for Bioanalysis Studies of a Cell Therapy Product with a Special Focus on Matrix Interferences in a Wide Range of Organ Tissues.

Article

Author: Norrick, Alexandra ; Frank, Markus H ; Ganss, Christoph ; Kluth, Mark A ; Niebergall-Roth, Elke ; Esterlechner, Jasmina ; Schröder, Hannes M

Quantitative polymerase chain reaction (qPCR) has emerged as an important bioanalytical method for assessing the pharmacokinetics of human-cell-based medicinal products after xenotransplantation into immunodeficient mice. A particular challenge in bioanalytical qPCR studies is that the different tissues of the host organism can affect amplification efficiency and amplicon detection to varying degrees, and ignoring these matrix effects can easily cause a significant underestimation of the true number of target cells in a sample. Here, we describe the development and drug regulatory-compliant validation of a TaqMan^® qPCR assay for the quantification of mesenchymal stromal cells in the range of 125 to 20,000 cells/200 µL lysate via the amplification of a human-specific, highly repetitive α-satellite DNA sequence of the chromosome 17 centromere region HSSATA17. An assessment of matrix effects in 14 different mouse tissues and blood revealed a wide range of spike recovery rates across the different tissue types, from 11 to 174%. Based on these observations, we propose performing systematic spike-and-recovery experiments during assay validation and correcting for the effects of the different tissue matrices on cell quantification in subsequent bioanalytical studies by multiplying the back-calculated cell number by tissue-specific factors derived from the inverse of the validated percent recovery rate.

01 Jul 2023·Cytotherapy

ABCB5+ mesenchymal stromal cells facilitate complete and durable wound closure in recessive dystrophic epidermolysis bullosa

Article

Author: Dieter, Kathrin ; Tolar, Jakub ; Niebergall-Roth, Elke ; Kluth, Mark A ; Frank, Natasha Y ; Fluhr, Silvia ; McGrath, John A ; Frank, Markus H ; Kiritsi, Dimitra ; Ganss, Christoph ; Daniele, Cristina

BACKGROUND AND AIMSRecessive dystrophic epidermolysis bullosa (RDEB) is a hereditary, rare, devastating and life-threatening skin fragility disorder with a high unmet medical need. In a recent international, single-arm clinical trial, treatment of 16 patients (aged 6-36 years) with three intravenous infusions of 2 × 10⁶ immunomodulatory ABCB5⁺ dermal mesenchymal stromal cells (MSCs)/kg on days 0, 17 and 35 reduced disease activity, itch and pain. A post-hoc analysis was undertaken to assess the potential effects of treatment with ABCB5⁺ MSCs on the overall skin wound healing in patients suffering from RDEB.METHODSDocumentary photographs of the affected body regions taken on days 0, 17, 35 and at 12 weeks were evaluated regarding proportion, temporal course and durability of wound closure as well as development of new wounds.RESULTSOf 168 baseline wounds in 14 patients, 109 (64.9%) wounds had closed at week 12, of which 63.3% (69 wounds) had closed already by day 35 or day 17. Conversely, 74.2% of the baseline wounds that had closed by day 17 or day 35 remained closed until week 12. First-closure ratio within 12 weeks was 75.6%. The median rate of newly developing wounds decreased significantly (P = 0.001) by 79.3%.CONCLUSIONSComparison of the findings with published data from placebo arms and vehicle-treated wounds in controlled clinical trials suggests potential capability of ABCB5⁺ MSCs to facilitate wound closure, prolongate wound recurrence and decelerate formation of new wounds in RDEB. Beyond suggesting therapeutic efficacy for ABCB5⁺ MSCs, the analysis might stimulate researchers who develop therapies for RDEB and other skin fragility disorders to not only assess closure of preselected target wounds but pay attention to the patients' dynamic and diverse overall wound presentation as well as to the durability of achieved wound closure and the development of new wounds.TRIAL REGISTRATIONClinicaltrials.gov NCT03529877; EudraCT 2018-001009-98.

100 Deals associated with RHEACELL GmbH & Co. KG

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100 Translational Medicine associated with RHEACELL GmbH & Co. KG

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Pipeline Snapshot as of 23 Nov 2024

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3

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