Jyou to fine fraction our 20,000 genes may cause disease when destroyed, but this sliver accounts for thousands of rare diseases. The difficulty is, what can a doctor do to cure them? With a disease as common as type 2 diabetes, the underlying biology is the same across millions of patients. The doctor may prescribe metformin. But with a genetic disease, the mutation may only affect a small number of people around the world. In many cases doctors won't even know which mutation is to blame, let alone how to fix it.
New breakthroughs in gene editing headers. But treatments are expensive and difficult to develop. The cost of delivering any new drug to patients is now about $2 billion, in part because, as Brian David Smith notes in New medicines, fair prices“the success rate from discovery to market is negligible,” and there are approved treatments for “less than 10% of the 8,000 diseases that affect humans.” Commercial incentives, he argues, distort innovation toward profitable cancer drugs and long-term treatments for large populations. Comprehensive gene therapies for very rare diseases are considered too expensive to develop and too low to generate profit.
Let's take 2022 revolutionary gene therapy to treat a young patient with relapsed T-cell leukemia at Great Ormond Street Hospital (GOSH) in London. Wasim Qasim, the pediatric immunologist leading this work, warned Earlier this year we said that “we'll end up with a treatment that works but that no one wants to pay for.” Sustaining innovative work cannot rely on philanthropy alone. His technological achievement – “off-the-shelf” edited T cells – will require widespread access to industrial production, and the market alone will not create it. GOSH research hints at the promise—and limitations—of therapy.
The money from the biotech boom has moved into silicon chips and artificial intelligence. That's why a major milestone in primary editing—DNA search-and-replace technology—was announced with a warning this May. Therapeutics company Prime Medicine successfully treated a teenager with a rare immune disease, but… said it would stop future work. The geopolitics of big pharma are also changing. Firms are moving research and production to countries with large and long-term political support, such as China and the United States. Britain looks vulnerable. Donald Trump has already forced the UK to agree to higher prices for NHS drugs under the threat of tariffs. This means less money for patient support and breakthrough treatments.
Gene therapy costs per patient can reach six or seven digits. If the drug exists but the business model does not, the latter must change. The UK needs state-backed manufacturing for medical sovereignty – although it should not ignore the reality that China can already produce some gene therapies much cheaper. What is needed is a renewed social contract in which innovation is diffused rather than hoarded. Gene therapy for rare diseases can be thought of as dialysis or transplantation: a collectively funded NHS service rather than a market commodity.
Many experimental treatments can remain part of long-term NHS research programs and be offered at cost while the science develops. Only a universal, government-funded health care system – with fair access and lifelong follow-up—could make gene therapy a drug rather than a commodity. Without the NHS and charities, discoveries will remain in the laboratory. This makes them part of the common good.
