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Treatment · Inherited blindness

EDIT-101 (Brilliance)

The first CRISPR therapy injected directly into a human organ — scientifically encouraging, and discontinued because the eligible population was too small.

Discontinued in vivoeyediscontinuedCEP290
Clinical research Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.

Explicación sencilla

EDIT-101 was injected under the retina to cut out a faulty piece of a gene causing a severe inherited blindness. Some patients saw meaningfully better. The programme was then stopped — not because it failed, but because so few people in the world have that exact mutation that developing it further did not make commercial sense. It is the clearest example in the field of good science ending for economic reasons.

Profundizar

EDIT-101 delivered SpCas9 and dual guides by subretinal AAV5 injection to excise the intronic CEP290 c.2991+1655A>G variant causing Leber congenital amaurosis type 10. The Phase 1/2 BRILLIANCE trial reported clinically meaningful vision improvement in a subset of participants with an acceptable safety profile, published in the New England Journal of Medicine. Editas discontinued development, citing the small eligible population, and subsequently reset its pipeline around in vivo cardiovascular targets.

Sources

Written by The CRISPR Atlas editorial team Last updated Aug 25, 2026

Connected in the Atlas

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Technologies

CRISPR-Cas9

Diseases

Inherited Blindness

Clinical Trials

Single Ascending Dose Study of EDIT-101 in Participants With Leber Congenital Amaurosis Type 10

Genes

CEP290

Companies

Editas Medicine
Solo información educativa Esta página es una referencia, no asesoramiento médico. El estado de la investigación y la situación regulatoria cambian; comprueba la fecha de última actualización que aparece arriba y confirma cualquier dato importante con las fuentes primarias indicadas.