- Editas Medicine and partner Allergan on Thursday opened enrollment in what they say is the world's first study testing a CRISPR-based therapy that works by modifying genes inside the human body.
- The Phase 1/2 trial, dubbed BRILLIANCE, will involve 18 patients with an inherited form of blindness known as Leber congenital amaurosis 10, or LCA10. Mutations in the CEP290 gene lead to progressive loss of photoreceptor cells, which in turn causes vision to deteriorate.
- Editas proposes to fix that with EDIT-101, which is given via an injection directly into the eye. The CRISPR-based medicine cuts out the relevant DNA, hopefully restoring normal protein expression in the process.
Nearly six years after launching on the promise of editing DNA, Editas is beginning the first human test of whether its CRISPR-based approach can live up to its unparalleled promise.
Editas and partner Allergan's milestone follows other firsts for the gene-editing field: Sangamo Therapeutics has tested its older zinc finger technology in patients with Hunter and Hurler syndromes, while CRISPR Therapeutics and Vertex treated the first individual with a CRISPR-based therapeutic earlier this year.
The difference between what Editas and Allergan plan to do and the study already begun by CRISPR and Vertex is where the intended gene editing takes place. Editas' medicine cuts DNA inside the body, while CRISPR uses the gene-editing technology to engineer stem cells extracted from patients.
Gene therapy delivered by viral vectors has already proven successful in eye disease. Spark Therapeutics' Luxturna treats a similar disease to what Editas and Allergan are targeting, but caused by mutations in the RPE65 gene.
For BRILLIANCE, Editas and Allergan will test EDIT-101, now also called AGN-151587, in as many as five patient cohorts across three ascending dose levels. Patients from ages 3 to 17 will be given the therapy in one eye.
The companies plan to enroll study participants through four trial sites, including the Massachusetts Eye and Ear center.
Enrollment is important, as LCA10 is exceedingly rare, affecting between two and three people out of every 100,000 born, according to Editas. The companies expect the first patient to be dosed in the second half of this year.
Launch of BRILLIANCE comes seven months after Editas' CEO, Katrine Bosley, stepped down after four and a half years as company head. Her departure followed those of the biotech's chief medical and financial officers, raising some concerns about the biotech's leadership at a critical time for its pipeline.
Cynthia Collins has been serving as interim CEO in Bosley's place since February.