One-Shot CRISPR Could Rewrite Blood Disorder Care

Editas’ single-dose CRISPR therapy could make sickle cell treatment faster, cheaper, and far more accessible.

24 Jun 2025

At a conference in Milan on June 12th, Editas Medicine, a gene-editing firm, unveiled an approach that could make treating inherited blood disorders much simpler. Instead of harvesting a patient’s stem cells, editing them in a lab and then reinfusing them, a costly, weeks-long process, the company proposes delivering CRISPR tools directly into bone marrow cells via a single injection.

The method uses lipid nanoparticles, tiny fat-based capsules, to carry the editing machinery in vivo. In tests on non-human primates, a single dose produced efficient genetic edits in most targeted blood-forming cells. The changes switched on fetal haemoglobin, which can compensate for defective adult haemoglobin found in sickle-cell disease and beta-thalassemia. Early results suggest the editing rates exceed the threshold thought necessary for clinical benefit.

“Our goal is to make durable gene editing available to patients everywhere, not just in elite medical centers,” said Dr Linda C. Burkly, the firm’s chief scientific officer.

By avoiding the use of viral vectors, which are expensive to make and pose some safety risks, the platform could cut manufacturing costs and speed development. It would also reduce dependence on specialist hospital infrastructure, widening access in poorer or rural areas. If it works in humans, the approach could change how the industry develops CRISPR-based therapies, steering rivals towards similar non-viral delivery systems and encouraging regulators to adapt approval pathways.

Much still depends on trials in people, which have yet to begin. Delivery has long been the bottleneck for in vivo gene editing. If Editas’s nanoparticles can consistently breach it, the company may have moved the prospect of a one-shot genetic cure for sickle-cell disease from a distant ambition to an imminent possibility.