Eye On the Cure Research News

Jul 25, 2019

Allergan and Editas Begin Recruiting for CRISPR/Cas9 Clinical Trial for LCA10

The trial will be the first for a CRISPR/Cas9 therapy administered inside the human body.

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Allergan, a global pharmaceutical company, and Editas Medicine, a developer of gene-editing therapies, have begun patient recruitment for a Phase 1/2 clinical trial for a CRISPR/Cas9 treatment for people with Leber congenital amaurosis 10 (LCA10). The treatment targets a specific mutation (c.2991+1655A>G in Intron 26) of the gene CEP290. 

Known as the Brilliance clinical trial, the study is the first for a CRISPR/Cas9 treatment for an inherited retinal disease. It is also the first clinical trial for a CRISPR/Cas9 therapy administered inside the human body.

Brilliance is a dose escalation trial that will enroll adult and pediatric (3 – 17 years of age) patients in the US. Massachusetts Eye and Ear (Boston) is currently enrolling patients. 

The launch of the Brilliance clinical trial is an exciting milestone for people with inherited retinal diseases. CRISPR/Cas9 is potentially powerful technology for correcting a mutated gene so that it expresses a functional protein and provides better vision to the patient

Benjamin Yerxa, PhD, chief executive officer at the Foundation Fighting Blindness

Known as EDIT-101, the CRISPR/Cas9 gene-editing technology developed by Allergan and Editas is designed is to locate and remove the mutation in LCA10. The treatment works like a pair of molecular scissors to cut out the mutation. The treatment is delivered to photoreceptors by a subretinal injection.

Gene editing is different from gene (replacement) therapy. In gene therapy, copies of an entirely new gene are delivered to the retina to replace the defective copies. In CRISPR/Cas9 gene editing, only the mutated region of the gene is corrected.

“The launch of the Brilliance clinical trial is an exciting milestone for people with inherited retinal diseases. CRISPR/Cas9 is potentially powerful technology for correcting a mutated gene so that it expresses a functional protein and provides better vision to the patient,” says Benjamin Yerxa, PhD, chief executive officer at the Foundation Fighting Blindness. “CRISPR/Cas9 treatments can be especially desirable when gene therapy isn’t feasible because the large replacement gene — for example, CEP290 — exceeds the capacity of commonly used viral gene delivery systems such as adeno-associated viruses or AAVs.”

Learn more about the Foundation’s CRISPR/Cas9 activities from Dr. Steve Rose.