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News of a ‘CRISPR baby’ broke this week, almost seven years after the first CRISPR baby scandal unleashed a media frenzy and years of ethical debates. But this time, the story marks a very different chapter in the evolution of human gene editing. Rather than an unregulated experiment carried out in secrecy, this new case involved a safe, custom-designed CRISPR therapy, developed with clinical oversight, to treat a life-threatening genetic disorder in a newborn. The therapy targeted a disease-causing mutation in the infant’s DNA and was administered after birth, not at the embryonic stage.
The child, who had been unable to properly metabolize protein due to a rare genetic condition, received three doses of the CRISPR treatment in early 2025. The therapy was created in just six months by a collaborative team of researchers and clinicians from academia, industry, and government. According to treating physicians, the infant is now growing and developing well. Although it’s too early to say whether the treatment is curative, the early signs are promising.
This case is not about germline editing or enhancement. It is about safety, precision, and therapeutic intervention, carried out in a way that aligns with regulatory frameworks and ethical principles. One of the most fundamental ethical failures of the original CRISPR baby case was not just the secrecy or lack of informed consent, but the premature use of an innovative technology before it was ready. At the time, CRISPR had not been adequately characterized; its off-target effects, long-term safety, and biological complexity were still poorly understood.
By contrast, this recent intervention reflects a field that has matured. It builds on nearly a decade of scientific progress, rigorous clinical trials, and global regulatory evolution. When used responsibly, gene editing can deliver meaningful, targeted, and life-saving therapies. This raises an important question: how far have we come since the first CRISPR-edited babies, and where does the field stand today?
Evolution of CRISPR technology and applications
Over the past decade, CRISPR has moved from controversy to clinic. The field has evolved rapidly, from early ex vivo trials to in vivo delivery, base editing, and now bespoke therapies for individual patients. To provide an overview of this evolution, we created a timeline of every publicly reported therapeutic use of CRISPR in humans. It is important to note that various gene editing experiments have been conducted in vitro and in viable embryos that were not subsequently implanted as this remains illegal.
Across these studies, CRISPR has shown remarkable potential, particularly for monogenic diseases where a single faulty gene can be targeted and corrected. Conditions like sickle cell disease, beta-thalassemia, and hereditary blindness have seen the most success, with patients often experiencing durable improvements or remission. Even early in vivo trials have demonstrated encouraging safety profiles, with few adverse events and strong biological responses. Since 2023, various CRISPR therapies have been approved globally for these conditions. A comprehensive list of current CRISPR-based clinical trials can be found here.
Scientific and economic barriers
Despite remarkable progress, significant challenges remain. Clinical trials targeting cancer have shown mixed results, often due to the inherent complexity and variability of solid tumors, which make them more difficult to treat with precision-based approaches. Multifactorial diseases also remain beyond the current reach of gene-editing technologies, as most CRISPR strategies are designed to target single-gene disorders rather than conditions shaped by a combination of genetic and environmental factors. Perhaps most critically, the cost of CRISPR therapies continues to pose a significant barrier. With treatments priced between $370K and $3.5 million per patient, accessibility is severely limited. As these innovative treatments continue to be developed and receive regulatory approval, it is imperative to tackle questions about equity.
Addressing these disparities will require a collective effort from scientists, biotech and pharma companies, governments, insurers, global health organizations, and patient advocacy groups. Coordination and collaboration across diseases and targets can help reduce redundancy and enhance efficiency, which may accelerate therapies and reduce costs. Some strategies may include:
- Public–private partnerships: Joint initiatives between biotech or pharma companies and national health systems could subsidize or co-develop therapies in high-need regions.
- Technology transfer & local manufacturing: Enabling regional hubs for CRISPR therapy development in low- to middle-income countries could reduce costs and decentralize access.
- Tiered pricing models: Adaptive pricing based on local economic context could lower the financial barriers for eligible patients.
- Open innovation frameworks: Sharing early-stage development tools, delivery platforms, or manufacturing standards.
- Clinical trial equity: Ensuring diverse representation in trials from the start is essential for maintaining both scientific validity and inclusive access.
Rethinking the Boundaries of Gene Editing
Of note, embryonic genome editing remains prohibited worldwide. While the science has advanced, public understanding hasn’t always kept pace. Misconceptions about what CRISPR can and can’t do continue to shape popular opinion. The idea of “designer babies” is often raised in public discourse, despite the fact that any clinical use of gene editing, particularly in embryos, would be tightly regulated and restricted to treating serious, life-threatening conditions. In reality, the kind of targeted, well-controlled gene editing used in current therapies bears little resemblance to the speculative enhancements that dominate cultural anxieties.
Resistance to gene editing also arises from philosophical, religious, and ethical worldviews, many of which understandably question how far science should go in altering human biology. These perspectives are important, and they deserve space in the conversation. But they can also contribute to a blanket suspicion of the technology, even when it is used in ways that are cautious, transparent, and life-saving.
In a recent episode of The Genetics Podcast, we spoke with Helen O’Neill, a leading expert in prenatal genetics, about how the 2018 CRISPR baby case and subsequent sensational media headlines have cast a persistent and dark shadow across the field, with public opinion remaining unchanged despite significant progress in technology.
As gene editing enters a new era defined by real patients, regulated therapies, and scientific maturity, it’s clear that the latest developments are not a repeat of past controversies. The path forward depends not only on innovation, but also on clear communication, ethical transparency, and global collaboration. This will ensure that what’s possible in the lab translates into what’s acceptable, equitable, and understood in society.
Check out the podcast episode on your platform of choice below or read the recap blog.
📽️ EP 187: Rethinking germline gene editing and revolutionizing women’s fertility with Helen O’Neill