Formulation of Cell-Based Medicinal Products: A Question of Life or Death?

Karin H Hoogendoorn; Daan J A Crommelin; Wim Jiskoot

doi:10.1016/j.xphs.2020.07.002

Formulation of Cell-Based Medicinal Products: A Question of Life or Death?

Karin H Hoogendoorn, Daan J A Crommelin, Wim Jiskoot

Leiden University Medical Center, Hospital Pharmacy, Interdivisional GMP Facility, Albinusdreef 2, 2333 ZA Leiden, the Netherlands.
Division of BioTherapeutics, Leiden Academic Centre for Drug Research (LACDR), Leiden University, Einsteinweg 55, 2333 CC Leiden, the Netherlands. Electronic address: [email protected].

Research output: Contribution to journal › Comment/Letter to the editor › Academic › peer-review

Abstract

The formulation of cell-based medicinal products (CBMPs) poses major challenges because of their complexity, heterogeneity, interaction with their environment (e.g., the formulation buffer, interfaces), and susceptibility to degradation. These challenges can be quality, safety, and efficacy related. In this commentary we discuss the current status in formulation strategies of off-the-shelf and non-off-the-shelf (patient-specific) CBMPs and highlight advantages and disadvantages of each strategy. Analytical tools for the characterization and stability assessment of CBMP formulations are addressed as well. Finally, we discuss unmet needs and make some recommendations regarding the formulation of CBMPs.

Original language	English
Pages (from-to)	1885-1894
Number of pages	10
Journal	Journal of Pharmaceutical Sciences
Volume	110
Issue number	5
Early online date	7 Jul 2020
DOIs	https://doi.org/10.1016/j.xphs.2020.07.002
Publication status	Published - 1 May 2021

Keywords

Advanced therapy medicinal products
Biopharmaceuticals
Cell therapy products
Cell-based medicinal products
Excipients
Formulation
Fresh
Frozen
(Non)-off-the shelf
Stability

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10.1016/j.xphs.2020.07.002

PIIS0022354920303646Final published version, 308 KBLicence: Taverne

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title = "Formulation of Cell-Based Medicinal Products: A Question of Life or Death?",

abstract = "The formulation of cell-based medicinal products (CBMPs) poses major challenges because of their complexity, heterogeneity, interaction with their environment (e.g., the formulation buffer, interfaces), and susceptibility to degradation. These challenges can be quality, safety, and efficacy related. In this commentary we discuss the current status in formulation strategies of off-the-shelf and non-off-the-shelf (patient-specific) CBMPs and highlight advantages and disadvantages of each strategy. Analytical tools for the characterization and stability assessment of CBMP formulations are addressed as well. Finally, we discuss unmet needs and make some recommendations regarding the formulation of CBMPs.",

keywords = "Advanced therapy medicinal products, Biopharmaceuticals, Cell therapy products, Cell-based medicinal products, Excipients, Formulation, Fresh, Frozen, (Non)-off-the shelf, Stability",

author = "Hoogendoorn, {Karin H} and Crommelin, {Daan J A} and Wim Jiskoot",

year = "2021",

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doi = "10.1016/j.xphs.2020.07.002",

language = "English",

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TY - JOUR

T1 - Formulation of Cell-Based Medicinal Products

T2 - A Question of Life or Death?

AU - Hoogendoorn, Karin H

AU - Crommelin, Daan J A

AU - Jiskoot, Wim

PY - 2021/5/1

Y1 - 2021/5/1

N2 - The formulation of cell-based medicinal products (CBMPs) poses major challenges because of their complexity, heterogeneity, interaction with their environment (e.g., the formulation buffer, interfaces), and susceptibility to degradation. These challenges can be quality, safety, and efficacy related. In this commentary we discuss the current status in formulation strategies of off-the-shelf and non-off-the-shelf (patient-specific) CBMPs and highlight advantages and disadvantages of each strategy. Analytical tools for the characterization and stability assessment of CBMP formulations are addressed as well. Finally, we discuss unmet needs and make some recommendations regarding the formulation of CBMPs.

AB - The formulation of cell-based medicinal products (CBMPs) poses major challenges because of their complexity, heterogeneity, interaction with their environment (e.g., the formulation buffer, interfaces), and susceptibility to degradation. These challenges can be quality, safety, and efficacy related. In this commentary we discuss the current status in formulation strategies of off-the-shelf and non-off-the-shelf (patient-specific) CBMPs and highlight advantages and disadvantages of each strategy. Analytical tools for the characterization and stability assessment of CBMP formulations are addressed as well. Finally, we discuss unmet needs and make some recommendations regarding the formulation of CBMPs.

KW - Advanced therapy medicinal products

KW - Biopharmaceuticals

KW - Cell therapy products

KW - Cell-based medicinal products

KW - Excipients

KW - Formulation

KW - Fresh

KW - Frozen

KW - (Non)-off-the shelf

KW - Stability

U2 - 10.1016/j.xphs.2020.07.002

DO - 10.1016/j.xphs.2020.07.002

M3 - Comment/Letter to the editor

C2 - 32649938

SN - 0022-3549

VL - 110

SP - 1885

EP - 1894

JO - Journal of Pharmaceutical Sciences

JF - Journal of Pharmaceutical Sciences

IS - 5

ER -

Formulation of Cell-Based Medicinal Products: A Question of Life or Death?

Abstract

Keywords

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