Editas Medicine, Inc. (NASDAQ: EDIT), based in Dublin, Ireland and Cambridge, Massachusetts, and Allergan plc (NYSE: AGN), also headquartered in Dublin, Ireland have announced that the first potential patient has been successfully screened to administer EDIT-101 by early 2020. “EDIT-101” is a CRISPR-based technology for the treatment of Leber Congenital Amaurosis type 10 (LCA10). CRISPR (an acronym for “clustered regularly interspaced short palindromic repeats”) is one of the latest gene-editing tools, originally uncovered as a bacterial defense mechanism against phage infection, and now under development as a potential gene therapy approach by multiple biotech and pharmaceutical companies worldwide.
The core opportunity of CRISPR technology has been developed by a number of key biotech players, chiefly the founding scientists currently or previously associated with several start-up companies, including Crispr Therapeutics (Basel, Switzerland), Editas Medicine (Dublin, Ireland and Cambridge, Massachusetts), Caribou Biosciences, Inc. (Berkeley, California), Intellia Therapeutics (Cambridge, Massachusetts), and ERS Genomics (Dublin, Ireland). The technology derives from observations made in the repetitive sequences isolated from a number of prokaryotic and archaebacteria, first identified in 1987 by Yoshizumi Ishino and Atsuo Nakata, then at Japan’s Research Institute for Microbial Diseases, Osaka University. Working on the E.coli iap enzyme, responsible for the isozyme conversion of alkaline phosphatase, Ishino and colleagues reported an unusual set of 29 nucleotide repeats interspersed with five intervening 32 nucleotide sequences, seemingly without any discernible function. Over a period of 10+ years, as increasing numbers of DNA sequences were deposited in the public databases, and a growing number of similarly structured repeat sequences, interspersed with unusual sequence, were reported from several different bacterial and archael strains.
Currently, Editas’ and Allergan’s Phase 1/2 clinical trial of AGN-151587 (also referred to EDIT-101), is an experimental medicine under development for the treatment of LCA10, caused by mutations in the CEP290 gene. The clinical study is being carried out in three centres located in the US with an estimated enrolment of 18 patients (paediatric/adult). This trial is an open-label, single ascending dose study to evaluate the safety, tolerability, and efficacy of the gene therapy in patients to be eligible 3 years or older. Commenting on the announcement, Charles Albright, Ph.D., Chief Scientific Officer, Editas Medicine, stated that “now that enrolment is underway, we are one step closer to delivering a transformative medicine to LCA10 patients. The team at Editas looks forward to continuing to collaborate with our partners at Allergan, patient advocacy organizations, and the inherited retinal diseases community as we develop this and other durable experimental medicines for patients with devastating ocular diseases.”