Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
What this trial is testing
The first time CRISPR was injected directly into a human organ: under the retina, to remove a faulty piece of a gene causing severe inherited blindness. Some participants saw meaningfully better. The programme was later discontinued because too few people in the world have that exact mutation.
An open-label, single ascending dose Phase 1/2 study of EDIT-101, delivered by subretinal AAV5 injection, in participants with CEP290-associated Leber congenital amaurosis type 10. Published results reported clinically meaningful improvement in visual function in a subset of participants with an acceptable safety profile. Editas subsequently discontinued development for population-size reasons.
Sources
- New England Journal of Medicine · 2024
Gene editing for CEP290-associated retinal degeneration ↗
What a trial result does and does not mean
Early-phase results come from small numbers of participants and short follow-up. They can change with more people and more time, and a trial that is recruiting, active or even completed has not established that a treatment works. Enrolment criteria are strict; eligibility is decided by the trial site, never by a website.