Editas Medicine, a company developing gene-editing treatments, has received authorization from the US Food and Drug Administration to launch a clinical trial for its emerging CRISPR/Cas9 therapy for people with a mutation in the gene CEP290, which causes Leber congenital amaurosis 10 (LCA10). LCA causes severe vision loss or blindness at birth.
Known as EDIT-101, the treatment will be evaluated in a Phase 1/2, dose-escalation clinical trial. The company expects to enroll 10 to 20 patients. Further details and contact information for the study will be listed soon at www.clinicaltrials.gov. EDIT-101 targets a specific mutation, “c.2991+1655A>G” in intron 26, of the CEP290 gene.
Editas has earned a $25 million milestone payment from Allergan as part of a drug-development alliance.
“We are very pleased with this historic milestone in the treatment of inherited retinal diseases,” says Stephen Rose, PhD, chief scientific officer, Foundation Fighting Blindness. “EDIT-101 is the first gene-editing therapy using CRISPR/Cas9 to move into a human study for a retinal disease. The approach shows great promise for treating these vision-robbing diseases.”
CRISPR/Cas9 is the acronym for: Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-associated 9. The technology is engineered to locate the disease-causing genetic defect. It then works like molecular scissors to cut out the mutated portion of the gene. In some cases, it also inserts a healthy piece of DNA.
CRISPR/Cas9 is different from gene therapy. In gene therapy, copies of a whole new gene are delivered to replace the defective copies of the gene. CRISPR/Cas9, on the other hand, is a “cut-and-paste” approach to fixing the patient’s own mutated gene.
One big advantage of CRISPR is getting around the problem of delivering large replacement genes — CEP290 and USH2A, for example — that won’t fit in the human-engineered viruses designed to carry them into retinal cells.
You can learn about Foundation-funded CRISPR/Cas9 projects by going to the blog post: ARVO 2018: Dr. Steve Rose Reports on CRISPR/Cas9 for Inherited Retinal Diseases.