Gene-editing technology holds extraordinary promise for people living with rare and often devastating genetic disorders. In some cases, a single precise edit to DNA could permanently correct the underlying cause of disease. Yet despite these scientific breakthroughs, most patients never see a treatment reach the clinic. The obstacle is not biology, but economics.
Developing a gene-editing therapy is an expensive and complex process, often costing millions of dollars from early research through manufacturing and clinical delivery. For pharmaceutical companies, those costs are difficult to justify when a treatment may be needed by only one patient or a handful worldwide. As a result, many rare conditions — particularly ultra-rare diseases — remain largely ignored by traditional drug development models.
David Liu, a pioneering scientist in gene editing and a Nobel Prize recipient, argues that the problem can be addressed by fundamentally changing how these therapies are created. Rather than relying on a highly customized, step-by-step approach where each phase is designed independently and billed separately, Liu and his colleagues believe a more integrated system could dramatically reduce costs and timelines.
This thinking has led to the creation of the Center for Genetic Surgery, a new nonprofit initiative based at the Broad Institute, which is affiliated with Harvard University. The center aims to bring together all key participants in the gene-editing process — including patients, clinicians, researchers, manufacturers, and biotech companies — under a single collaborative framework.
By coordinating these groups from the outset, the center hopes to standardize and streamline many of the steps that currently drive up costs. Manufacturing processes, regulatory pathways, and clinical delivery could be simplified and reused across multiple treatments, rather than rebuilt from scratch each time. In today’s system, bespoke therapies can carry price tags exceeding $3 million per patient, making them inaccessible to most health systems.
Supporters of the collaborative model argue that greater efficiency could unlock the full potential of gene editing, allowing science to serve patients who have long been overlooked. Instead of focusing only on conditions with large markets, researchers could address the “long tail” of rare diseases that collectively affect millions of people worldwide.
The Center for Genetic Surgery represents a significant departure from the fragmented structure of modern health care, where innovation, manufacturing, and treatment are often siloed and driven by separate financial incentives. If successful, the model could offer a blueprint for making highly personalized medicine both scalable and sustainable.
While challenges remain — including regulatory hurdles and funding constraints — the initiative reflects a growing recognition that breakthrough science alone is not enough. To truly transform lives, gene-editing technologies must be paired with new ways of organizing care, sharing risk, and aligning incentives across the entire medical ecosystem.