Translating Cell and Gene Biopharmaceutical Products for Health and Market Impact. Product Scaling From Clinical to Marketplace: Lessons Learned and Future Outlook

Bak, Annette; Friis, Kristina Pagh; Wu, Yan; Ho, Rodney J. Y.

doi:10.1016/j.xphs.2019.05.027

Cited by 15 publications

(2 citation statements)

References 14 publications

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“…Here, the validation of the manufacture process and QC for batch release of the new ATMP tIPE has been described. The specific challenges included the following: setting the GMP requirements, standardization, assessment of its biological nature, short production times, potential alterations of the ATMP ex vivo (e.g., cell death), small sized autologous products whose amount is limited to clinical dose, irreversible cell labelling preventing cell characterization or purification, and the maintenance of a closed, GMP-compliant circuit from starting material collection to production, transport, and transplantation [ 9 , 34 ].…”

Section: Discussionmentioning

confidence: 99%

GMP-Grade Manufacturing and Quality Control of a Non-Virally Engineered Advanced Therapy Medicinal Product for Personalized Treatment of Age-Related Macular Degeneration

et al. 2022

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The introduction of new therapeutics requires validation of Good Manufacturing Practice (GMP)-grade manufacturing including suitable quality controls. This is challenging for Advanced Therapy Medicinal Products (ATMP) with personalized batches. We have developed a person-alized, cell-based gene therapy to treat age-related macular degeneration and established a vali-dation strategy of the GMP-grade manufacture for the ATMP; manufacturing and quality control were challenging due to a low cell number, batch-to-batch variability and short production duration. Instead of patient iris pigment epithelial cells, human donor tissue was used to produce the transfected cell product (“tIPE”). We implemented an extended validation of 104 tIPE productions. Procedure, operators and devices have been validated and qualified by determining cell number, viability, extracellular DNA, sterility, duration, temperature and volume. Transfected autologous cells were transplanted to rabbits verifying feasibility of the treatment. A container has been engineered to ensure a safe transport from the production to the surgery site. Criteria for successful validation and qualification were based on tIPE’s Critical Quality Attributes and Process Parameters, its manufacture and release criteria. The validated process and qualified operators are essential to bring the ATMP into clinic and offer a general strategy for the transfer to other manufacture centers and personalized ATMPs.

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Section: Discussionmentioning

confidence: 99%

GMP-Grade Manufacturing and Quality Control of a Non-Virally Engineered Advanced Therapy Medicinal Product for Personalized Treatment of Age-Related Macular Degeneration

et al. 2022

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“…Cell-based medicinal products (CBMPs) have recently become a key focus, with the initial market approvals of chimeric antigen receptor T cell therapy medicinal products in 2017 (127). Ex vivo genetically modified autologous CBMPs offer one of the most tailored patient-centric forms of medicinal products: A patient's cells are isolated, genetically modified, and infused back to the same patient.…”

Section: Cell-based Medicinal Productsmentioning

confidence: 99%

Patient Centricity Driving Formulation Innovation: Improvements in Patient Care Facilitated by Novel Therapeutics and Drug Delivery Technologies

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Khan

Kühl

et al. 2022

Annu. Rev. Pharmacol. Toxicol.

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Innovative formulation technologies can play a crucial role in transforming a novel molecule to a medicine that significantly enhances patients’ lives. Improved mechanistic understanding of diseases has inspired researchers to expand the druggable space using new therapeutic modalities such as interfering RNA, protein degraders, and novel formats of monoclonal antibodies. Sophisticated formulation strategies are needed to deliver the drugs to their sites of action and to achieve patient centricity, exemplified by messenger RNA vaccines and oral peptides. Moreover, access to medical information via digital platforms has resulted in better-informed patient groups that are requesting consideration of their needs during drug development. This request is consistent with health authority efforts to upgrade their regulations to advance age-appropriate product development for patients. This review describes formulation innovations contributingto improvements in patient care: convenience of administration, preferred route of administration, reducing dosing burden, and achieving targeted delivery of new modalities.

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Accelerating Drug Product Development and Approval: Early Development and Evaluation

et al. 2023

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Translating Cell and Gene Biopharmaceutical Products for Health and Market Impact. Product Scaling From Clinical to Marketplace: Lessons Learned and Future Outlook

Cited by 15 publications

References 14 publications

GMP-Grade Manufacturing and Quality Control of a Non-Virally Engineered Advanced Therapy Medicinal Product for Personalized Treatment of Age-Related Macular Degeneration

GMP-Grade Manufacturing and Quality Control of a Non-Virally Engineered Advanced Therapy Medicinal Product for Personalized Treatment of Age-Related Macular Degeneration

Patient Centricity Driving Formulation Innovation: Improvements in Patient Care Facilitated by Novel Therapeutics and Drug Delivery Technologies

Accelerating Drug Product Development and Approval: Early Development and Evaluation

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