Description: Discover the evolution of CRISPR, global milestones, regulatory challenges, and why Vietnam is becoming a strategic B2B hub for EU-GMP pharmaceutical manufacturing.
Keywords: gene editing technology, CRISPR-Cas9, base editing, prime editing, gene therapy, ATMP, EU-GMP Vietnam, CDMO Vietnam, pharmaceutical manufacturing, GMP-certified facilities
Introduction
In the past decade, gene editing has shifted from the realm of experimental science to one of the most promising fields in modern medicine. With the discovery of CRISPR-Cas9 in 2012, followed by the first regulatory approvals of CRISPR-based therapies in 2023–2024, the technology has reached a pivotal moment. For pharmaceutical companies, the rise of gene editing offers not only new therapeutic possibilities but also new challenges in manufacturing, scalability, and regulatory compliance.
As Southeast Asia—and Vietnam in particular—emerges as a hub for EU-GMP-certified pharmaceutical manufacturing, international partners are increasingly looking to the region for cost-efficient, compliant, and scalable solutions. This article explores the journey of gene editing, the milestones achieved, the leading nations driving innovation, and the future opportunities awaiting global pharmaceutical stakeholders in Vietnam.
A Brief History of Gene Editing
The story of modern gene editing began in 2012, when Emmanuelle Charpentier and Jennifer Doudna revealed that CRISPR-Cas9 could act as programmable “genetic scissors.” Their discovery earned them the 2020 Nobel Prize in Chemistry and laid the foundation for a revolution in molecular biology.
Soon after, the field expanded with the development of base editing (2016–2017), which allows researchers to correct single-letter DNA mutations without creating double-strand breaks. In 2019, scientists introduced prime editing, often described as a “search-and-replace” function for DNA, capable of inserting or rewriting genetic sequences with unprecedented precision.
By 2021, gene editing reached a new frontier when in vivo CRISPR therapy was tested in humans. Intellia Therapeutics’ NTLA-2001 demonstrated that a single IV infusion could reduce disease-causing proteins in patients with ATTR amyloidosis, proving that gene editing could work directly inside the human body.
Key Achievements
The most notable achievement in recent years is the approval of Casgevy (exa-cel), developed by Vertex Pharmaceuticals and CRISPR Therapeutics. In late 2023, the U.S. Food and Drug Administration (FDA) approved Casgevy for the treatment of sickle cell disease, and in early 2024, the therapy was also cleared for transfusion-dependent beta-thalassemia. The European Medicines Agency (EMA) followed with conditional marketing authorization, validating CRISPR-based therapy at a global level.
Alongside these regulatory milestones, in vivo applications have shown remarkable promise. NTLA-2001, for example, set a new benchmark for how gene editing can be delivered systemically through lipid nanoparticles. Meanwhile, base and prime editing technologies are evolving as second-generation tools, offering even greater precision and fewer risks of unintended changes.
These achievements collectively mark the transition of gene editing from theory to therapeutic reality—establishing it as one of the most significant medical advances of the 21st century.
Leading Nations in Gene Editing
Several countries have established themselves as leaders in the global gene-editing race.
The United States and the European Union remain at the forefront, driving innovation through clinical trials, regulatory frameworks, and funding ecosystems. The United Kingdom distinguished itself by being among the first to authorize CRISPR-based therapies for genetic blood disorders.
China has pursued a dual approach, investing heavily in medical applications while also leveraging gene editing in agriculture and food security. Its progressive regulatory stance in agriculture demonstrates a broad vision for gene editing beyond medicine.
In Southeast Asia, Singapore has taken a pioneering role by introducing the CTGTP (Cell, Tissue & Gene Therapy Products) regulatory framework, which clearly outlines clinical, manufacturing, and approval pathways for advanced therapies. This clarity has made Singapore an attractive base for global companies to launch trials and establish regional footprints.
Future Prospects
The next decade of gene editing promises to be transformative. One of the most important shifts will be the transition from ex vivo therapies—where patient cells are extracted, edited, and reinfused—to in vivo therapies, where editing occurs directly inside the patient’s body. This move could simplify treatment delivery, reduce costs, and expand access.
At the same time, next-generation editors such as base and prime editing are unlocking the ability to correct mutations once thought untreatable, while minimizing safety risks. The clinical pipeline is also broadening rapidly to include cardiovascular, neurological, and metabolic diseases, signaling the potential of gene editing to reshape entire areas of medicine.
These advances will inevitably create a rising demand for scalable, GMP-compliant manufacturing and strong partnerships between biotech innovators and global CDMOs.
Challenges in Mainstream Adoption
Despite its rapid progress, gene editing faces significant challenges before it can become a standard therapeutic option worldwide.
First, safety and specificity remain a scientific priority, as researchers continue to refine methods to avoid off-target edits and immune responses. Second, manufacturing complexity poses a barrier—gene therapies fall under the category of Advanced Therapy Medicinal Products (ATMPs), requiring rigorous GMP compliance and specialized infrastructure.
Economic barriers also play a role: with current therapies priced at more than USD 2 million per patient, healthcare systems must find sustainable models for reimbursement. Finally, regulatory and ethical concerns—particularly surrounding germline editing—place firm boundaries on what is permissible, ensuring that scientific ambition is balanced by societal responsibility.
Opportunities in Southeast Asia — Why Vietnam?
Conclusion
Gene editing has advanced rapidly from the discovery of CRISPR-Cas9 to the approval of the first therapies in the US and Europe. New approaches such as base editing and prime editing are expanding precision and safety, while different regions contribute with their own strengths—from regulatory frameworks to agricultural applications. Despite these breakthroughs, the field still faces challenges in safety, cost, and accessibility before gene editing can reach its full potential.
References
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Jinek, M., Chylinski, K., Fonfara, I., Hauer, M., Doudna, J. A., & Charpentier, E. (2012). A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity. Science, 337(6096), 816–821. (Discovery of CRISPR-Cas9)
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The Nobel Prize in Chemistry 2020. (2020). NobelPrize.org. (Awarded to Charpentier & Doudna for CRISPR-Cas9 development)
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Gaudelli, N. M., et al. (2017). Programmable base editing of A·T to G·C in genomic DNA without DNA cleavage. Nature, 551, 464–471. (Base editing technology)
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Anzalone, A. V., et al. (2019). Search-and-replace genome editing without double-strand breaks or donor DNA. Nature, 576, 149–157. (Prime editing technology)
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Gillmore, J. D., et al. (2021). CRISPR–Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis. New England Journal of Medicine, 385(6), 493–502. (In vivo CRISPR trial, NTLA-2001)
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U.S. Food and Drug Administration (FDA). (2023–2024). Approval of Casgevy (exa-cel) for sickle cell disease and beta-thalassemia. (CRISPR therapeutic approval)
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European Medicines Agency (EMA). (2024). Conditional Marketing Authorization for Casgevy. (EU regulatory milestone)
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Health Sciences Authority (HSA), Singapore. (2021). Regulatory overview of the CTGTP framework. (Singapore’s regulatory pathway for cell & gene therapies)