Investigating cell, tissue and gene therapy products and their regulation

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Cell, tissue and gene therapy products or (CTGTPs) are often breakthrough therapies with immense potential in treating diseases with no cure or rare diseases with high treatment burdens. However, few of such therapies have been approved and some have been recalled due to safety issues. Now, a study published in GaBI Journal [1] has investigated these therapies and the manufacturing and regulatory challenges they pose.

Autologous Stem Cells V13F14

The review highlights that CTGTPs are intended for use in humans for curative, prophylactic, palliative or diagnostic purposes and target diseases that are managed by therapies with high treatment burdens. CTGTPs include the use of stem cells for regenerative purposes, the insertion of genes related to the production of specific proteins in gene therapies for cancer and rare disease treatment, and the engineering of components involving cells and tissue architectures to mend and restore organs and tissues in tissue therapy. CTGTPs are also known as advanced therapy medicinal products or regenerative medicines.

CTGTPs are not conventional biologicals and are derived directly from individual patients or donors. To be produced, e.g. cells, tissues are required, whereas for conventional biological medicines the focus is on producing specific protein products. As such, storage, transport and cryopreservation of CTGTPs in the manufacturing cycle are key as cells require extreme cryopreservation temperatures, and heterogeneity has to be managed if the therapy product is autologous (from the patient). As a result, when compared to general biologicals, far more stringent processes are required in the manufacturing of CTGTPs. It is noted that some solutions to the challenges encountered in manufacturing include ensuring appropriate accreditation of professionals handling CTGTPs, using automation and outsourcing to contract development and manufacturing organizations to ensure better compliance to good manufacturing practice (GMP) standards.

The review elaborates that a great need for development of specific regulations and regulatory frameworks related to CTGTPs. It outlines that challenges faced in the manufacturing and regulation of CTGTPs include quality assurance issues, lack of expertise and limitations in technology. Overall, a global convergence in regulation related to CTGTPs is needed through global harmonization of information related to these innovative therapies. However, as they continue to evolve, regulation needs to be dynamic and responsive to advances. It is suggested that, to ensure these therapies are accessible to patients and have global reach, there needs to be greater and more effective communication between regulators and different stakeholders such as manufacturers, with feedback and some degree of flexibility incorporated into regulations.

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1. Sia CH, Koh C, Chan LW. Manufacture and regulation of cell, tissue and gene therapy products: global perspectives, challenges and next steps. Generics and Biosimilars Initiative Journal (GaBI Journal). 2022;11(2):65-81. doi:10.5639/gabij.2022.1102.012

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