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Abstract

Background

Current medicine could benefit from gene and cell therapies for genetic defects, cancer, and degenerative disorders. These therapies modify genetic material or biological components. CRISPR-Cas9 gene editing, stem cell, and CAR-T treatments are examples. Complex products need rigorous regulations to ensure quality, efficacy, and patient safety.

Objectives

This paper discusses international gene and cell-based treatment regulatory regimes, highlighting key issues and recent developments. It also includes gene and cell-based therapy classes and mechanisms.

Methods

The publications on gene and cell therapy challenges and their regulatory approvals in the US, Europe, Japan, Australia, Brazil, Canada, and China were collected over the last 20 years from PubMed, Scopus, and Google Scholar and analyzed to determine the differences.

Results

Gene treatments correct genetic defects or disease processes by adding, removing, or changing cell genetic information. In contrast, cell-based therapies restore damaged tissues with modified or unmodified cells. Highly customized and patient-specific drugs make regulatory monitoring challenging. US FDA CBER controls gene and cell-based therapies. Before clinical trials, these biologic drugs must file BLAs for market approval and INDs.

Discussion

FDA's Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations accelerate biological development. The EMA oversees EU Advanced Therapy Medicinal Products. ATMP quality, safety, and efficacy are CAT's top priorities. The Conditional Marketing Authorization process expedites access to life-threatening disease medicines while the MAA regulates them. Japan's PMDA's Conditional Time-Limited Approval for regenerative medicines provides early commercialization and rigorous post-market supervision. Similarly, each country has adopted some ways to expedite the approval of biologicals. Gene-editing drugs require specialized methods, long-term follow-up, and better safety to avoid off-target effects. GMPs ensure production uniformity, sterility, and safety, complicating manufacturing and quality control.

Conclusion

The review concludes that there is a need for worldwide regulatory harmonization and regulatory framework developments, including R.W.E., adaptive pathways, and personalization of biologics.

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2025-04-23
2025-09-03

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Challenges and Pathways in Regulating Next-Gen Biological Therapies
Bentham Science Publishers ; https://doi.org/10.2174/0113892010367028250411111549
/content/journals/cpb/10.2174/0113892010367028250411111549
/content/journals/cpb/10.2174/0113892010367028250411111549
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  • Article Type:     Research Article
Keywords: regulatory framework for biologicals ; Global regulation for biologicals ; Gene-based therapies ; Cell-based therapies

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