Gene editing breakthroughs offer unprecedented hope for treating thousands of rare genetic disorders, yet a fundamental flaw in the current system threatens to leave many patients behind. While advances like prime editing and “off-the-shelf” T-cell therapies show immense promise, the economics of drug development prioritize profitable treatments over rare-disease cures. This disconnect demands a new approach: a renewed social contract where innovation is shared, not dictated by market forces.
The Rare Disease Paradox
The human genome contains roughly 20,000 genes, but only a small fraction cause disease when disrupted. These mutations, while rare individually, collectively affect millions worldwide. However, treating genetic disorders differs drastically from common illnesses like type 2 diabetes. For widespread conditions, doctors can prescribe established treatments, but rare genetic mutations often affect so few people that developing a cure becomes financially unviable.
The cost of bringing a new drug to market now exceeds $2 billion, with less than 10% of known human diseases having approved treatments. As Brian David Smith notes, commercial incentives push innovation toward lucrative cancer drugs and chronic conditions, leaving rare-disease therapies underfunded. This isn’t a matter of scientific limitations; it’s an economic one.
The Limits of Philanthropy and Private Investment
Pioneering work, like the 2022 gene therapy at Great Ormond Street Hospital (GOSH) that treated a young leukemia patient, demonstrates the potential of new technologies. However, Waseem Qasim, the lead immunologist, warns that sustaining such advancements relies too heavily on philanthropy and unsustainable private investment.
Industrial-scale manufacturing is essential for wider availability, but the market alone won’t build it. Biotech companies are shifting focus to more profitable areas like AI and silicon chips, leaving critical gene therapy research stalled. Prime Medicine’s recent halt to further work on a prime editing breakthrough for a rare immune disorder underscores this trend.
Geopolitics and Medical Sovereignty
The landscape is further complicated by geopolitical shifts. Pharmaceutical firms are consolidating research and production in nations with robust policy support, like China and the US, while countries like Britain face increasing economic pressure.
Donald Trump’s trade tactics already forced the UK to accept higher drug prices, diverting funds from patient care and breakthrough therapies. Per-patient costs for gene therapies can reach seven figures, rendering them inaccessible without systemic change.
A New Social Contract
The current system is failing patients with rare genetic diseases. To address this, a fundamental shift in how we approach innovation is necessary. Treating gene therapy like essential services such as dialysis or transplants—funded collectively through an NHS-style model—would ensure access regardless of profitability.
While China’s lower manufacturing costs pose a competitive reality, Britain must pursue state-backed medical manufacturing to secure its own medical sovereignty. Innovation must be shared, not hoarded. The future of gene editing depends on redefining the relationship between science, business, and healthcare.
The existing structure ensures that cures remain out of reach for those who need them most. A renewed social contract—one that prioritizes human well-being over profit—is not just ethical; it’s essential.
