Futuristic Global Cell and Gene Therapy (CGT) Regulations Based on a Review of Today's Leading Frameworks
Hashmi, Shahrukh K Jamil, M Abdullah de la Fuente, Josu Mahlangu, Johnny Ozelo, Margareth C Walters, Mark C Almannaei, Asma Srivastava, Alok Alzahrani, Hazza Lu, Peihua
Hashmi, Shahrukh K
Jamil, M Abdullah
de la Fuente, Josu
Mahlangu, Johnny
Ozelo, Margareth C
Walters, Mark C
Almannaei, Asma
Srivastava, Alok
Alzahrani, Hazza
Lu, Peihua
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Computer Vision
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Journal article
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English
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Abstract
The advancement of Cell and Gene therapies (CGT) represents a pivotal shift in the treatment model for cancers and a broad spectrum of other diseases, particularly rare and orphan disorders. Despite major advances, information regarding global regulatory pathways is scarce and often perplexing. Essential technicalities to approval such as current Good Manufacturing Practice (cGMP), Good Laboratory Practices (GLP), Certificate of Pharmaceutical Product (CPP), and pathways that accelerate the approval process (like orphan drug designation) are often inadvertently marginalized or overcomplicated. Furthermore, the globalization of these therapies is crucially hampered by fragmented, economically unsustainable and unreliable regulatory pathways. These regulatory challenges and the high cost of the traditional clinical trial model create significant bottlenecks for developers. We analyze the current frameworks in major jurisdictions, highlighting the technicalities in each, and propose a new, harmonized global blueprint for development and approval. Key components of this proposed framework include a shift towards advanced preclinical models, adaptive trial design, and patient-centric endpoints. Along with simplifying the processes for CGT developers (whether industry or academia), adopting this framework could increase the efficiency of CGT approvals, and lower the development costs. Such changes would help in achieving the goal of bench to bedside to globalization, directly translating into a more robust research atmosphere that benefits all by increasing the efficiency of CGT trial and product approvals in both developed and developing countries.
Citation
S.K. Hashmi, M.A. Jamil, J. de la Fuente, J. Mahlangu, M.C. Ozelo, M.C. Walters , et al., "Futuristic Global Cell and Gene Therapy (CGT) Regulations Based on a Review of Today's Leading Frameworks," Cytotherapy, pp. 102064-102064, 2026, https://doi.org/10.1016/j.jcyt.2026.102064.
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Cytotherapy
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32 Biomedical and Clinical Sciences, 3206 Medical Biotechnology
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Elsevier