A risk-based approach for cell line development, manufacturing and characterization of genetically engineered, induced pluripotent stem cell–derived allogeneic cell therapies

Dashnau, Jennifer L.; Xue, Qunji; Nelson, Monica; Law, Eric; Cao, Liang; Hei, Derek J.

doi:10.1016/j.jcyt.2022.08.001

Cited by 9 publications

(3 citation statements)

References 70 publications

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“…Ensuring the safety and quality of iPSCs is imperative and strict quality control (QC) measures and adherence to Good Manufacturing Practices (GMP) are necessary to meet regulatory standards for clinical use. 4 , 5 In 2018, the Global Alliance for iPSC Therapies (GAiT) established guidelines for the QC of clinical-grade iPSC lines, with the aim of advancing iPSC-based treatments toward clinical reality while prioritizing safety, efficacy, and patient access. 6 Additionally, ethical considerations regarding the source of somatic cells, patient consent, and data privacy must be carefully addressed.…”

Section: Introductionmentioning

confidence: 99%

Manufacturing Parameters for the Creation of Clinical-Grade Human-Induced Pluripotent Stem Cell Lines From Umbilical Cord Mesenchymal Stromal Cells

Beckenkamp,

da Silva,

Von Hohendorff

et al. 2024

Stem Cells Translational Medicine

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Induced pluripotent stem cells (iPSCs) are reprogrammed cells with a remarkable capacity for unlimited expansion and differentiation into various cell types. Companies worldwide are actively engaged in developing clinical-grade iPSC lines to address the needs of regenerative medicine, immunotherapies, and precision medicine. However, ensuring the safety and quality of iPSCs is essential, with adherence to Good Manufacturing Practices (GMP) and ethical considerations being paramount. Perinatal cell and tissue banks, such as umbilical cord (UC) blood and tissue banks, are emerging as ideal sources for generating iPSCs due to their unique characteristics and GMP compliance. These banks provide access to immature cells with limited environmental exposure, known family and medical histories of donors, and readily available resources, thereby reducing the time and cost associated with personalized treatment strategies. This study describes the establishment of the first clinical-grade iPSC lines from umbilical cord mesenchymal stromal cells in Brazil. The process involved rigorous quality control measures, safety assessments, and adherence to regulatory standards, resulting in iPSCs with the necessary characteristics for clinical use, including sterility, genomic integrity, and stability. Importantly, the study contributes to the development of a Current Good Manufacturing Practice-compliant iPSC production pipeline in Brazil, using commercially available, chemically defined, and xeno-free products, along with validation by national outsourced laboratories, thereby facilitating the adoption of this technology within the country. The study emphasizes Brazil’s contribution to the progress of translational medicine and the promotion of scientific advancements within the field of regenerative and precision medicine.

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Section: Introductionmentioning

confidence: 99%

Manufacturing Parameters for the Creation of Clinical-Grade Human-Induced Pluripotent Stem Cell Lines From Umbilical Cord Mesenchymal Stromal Cells

Beckenkamp,

da Silva,

Von Hohendorff

et al. 2024

Stem Cells Translational Medicine

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“…In any event, the exclusion of genomic instability (via G-band karyotyping) and persistence of reprogramming systems (via quantitative PCR) are essential aspects of product characterization for cell-based therapies. 61 …”

Section: Introductionmentioning

confidence: 99%

Recent Progress in Retinal Pigment Epithelium Cell-Based Therapy for Retinal Disease

Klymenko,

González Martínez,

Zarbin

2024

Stem Cells Translational Medicine

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Age-related macular degeneration and retinitis pigmentosa are degenerative retinal diseases that cause severe vision loss. Early clinical trials involving transplantation of retinal pigment epithelial cells and/or photoreceptors as a treatment for these conditions are underway. In this review, we summarize recent progress in the field of retinal pigment epithelium transplantation, including some pertinent clinical trial results as well as preclinical studies that address issues of transplant immunology, cell delivery, and cell manufacturing.

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“…Effort has been focused on making post-mitotic, fully mature RPE cells from RPE stem cells present in adult cadaver eyes [ 28 ], iPSCs [ 29 ], and ESCs [ 4 ], reducing the risk for tumorgenicity. A risk-based approach guides decisions on the stage of development at which the cells should be transplanted into the eye [ 30 ].…”

Section: Introductionmentioning

confidence: 99%

Stem cell sources and characterization in the development of cell-based products for treating retinal disease: An NEI Town Hall report

et al. 2023

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National Eye Institute recently issued a new Strategic Plan outlining priority research areas for the next 5 years. Starting cell source for deriving stem cell lines is as an area with gaps and opportunities for making progress in regenerative medicine, a key area of emphasis within the NEI Strategic Plan. There is a critical need to understand how starting cell source affects the cell therapy product and what specific manufacturing capabilities and quality control standards are required for autologous vs allogeneic stem cell sources. With the goal of addressing some of these questions, in discussion with the community-at-large, NEI hosted a Town Hall at the Association for Research in Vision and Ophthalmology annual meeting in May 2022. This session leveraged recent clinical advances in autologous and allogeneic RPE replacement strategies to develop guidance for upcoming cell therapies for photoreceptors, retinal ganglion cells, and other ocular cell types. Our focus on stem cell-based therapies for RPE underscores the relatively advanced stage of RPE cell therapies to patients with several ongoing clinical trials. Thus, this workshop encouraged lessons learned from the RPE field to help accelerate progress in developing stem cell-based therapies in other ocular tissues. This report provides a synthesis of the key points discussed at the Town Hall and highlights needs and opportunities in ocular regenerative medicine.

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A risk-based approach for cell line development, manufacturing and characterization of genetically engineered, induced pluripotent stem cell–derived allogeneic cell therapies

Cited by 9 publications

References 70 publications

Manufacturing Parameters for the Creation of Clinical-Grade Human-Induced Pluripotent Stem Cell Lines From Umbilical Cord Mesenchymal Stromal Cells

Manufacturing Parameters for the Creation of Clinical-Grade Human-Induced Pluripotent Stem Cell Lines From Umbilical Cord Mesenchymal Stromal Cells

Recent Progress in Retinal Pigment Epithelium Cell-Based Therapy for Retinal Disease

Stem cell sources and characterization in the development of cell-based products for treating retinal disease: An NEI Town Hall report

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