Futuristic global cell and gene therapy regulations based on a review of today's leading frameworks

Authors

• Shahrukh K. Hashmi, Research and Innovation Center, UAE
• M Abdullah Jamil, Aga Khan University
• Josu de la Fuente, Imperial College London, UK
• Johnny Mahlangu, University of the Witwatersrand, South Africa
• Margareth C. Ozelo, State University of Campinas, Brazil
• Mark C. Walters, UCSF Benioff Children's Hospital Oakland, USA
• Asma Almannaei, Research and Innovation Center, UAE
• Alok Srivastava, St. John's National Academy of Health Sciences, India
• Hazza Alzahrani, King Faisal Specialist Hospital & Research Center, Saudi Arabia
• Peihua Lu, Lu Daopei Institute of Hematology, China
• Gili Kenet, Tel Aviv University, Israel
• Kaori Nomura, Fukushima Medical University, Japan
• Philippe Leboulch, Brigham & Women's Hospital and Harvard Medical School, USA

Document Type

Article

Department

Medical College Pakistan

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, good laboratory practices, critical process parameters 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 toward 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.

Publication (Name of Journal)

Cytotherapy

DOI

10.1016/j.jcyt.2026.102064

Recommended Citation

Hashmi, S. K., Jamil, M. A., Fuente, J. d., Mahlangu, J., Ozelo, M. C., Walters, M. C., Almannaei, A., Srivastava, A., Alzahrani, H., Lu, P., Kenet, G., Nomura, K., Leboulch, P. (2026). Futuristic global cell and gene therapy regulations based on a review of today's leading frameworks. Cytotherapy, 28(6), 1-13.
Available at: https://ecommons.aku.edu/pakistan_fhs_mc_mc/685