El Jian/Editas Start Ophthalmology CRISPR Therapy Phase I/II Clinical

ElJian and genome editing company Editas Medicine recently jointly announced that the ophthalmology gene editing therapy AGN-151587 (EDIT-101) Phase I/II clinical study BRILLIANCE (NCT03872479) has started patient entry groupAGN-151587 is being developed to treat Leiber Congenital Black Monsy 10, a genetic blindness caused by a mutation in the central protein 290 (CEP290) geneEditas Medicine was founded in 2013 by CRISPR "Big God" Zhang FengTHE BRILLIANCE STUDY IS THE COMPANY'S FIRST HUMAN CLINICAL STUDY IN ITS SIX YEARS, AND MARKS THE WORLD'S FIRST HUMAN CLINICAL STUDY OF CRISPR THERAPY FOR GENOME EDITING IN THE HUMAN BODYcrispR therapy, which has previously entered human clinical practice, is in vitro for genome editing, such as CRISPR Therapeutics and Vertex's CRISPR therapy CTX001 for beta thalassemiaThe genome editing of AGN-151587 is done in the human body, injecting under the retina into the eye photosensitive cells to eliminate the CEP290 gene mutation that causes LCA10so far, gene therapy based on viral vectors has been successful in treating hereditary eye diseaseSpark Therapeutics' one-time ophthalmology gene therapy Luxturna has been approved by the United States and the European Union to restore and improve vision for children and adults who have lost vision due to a mutation in the two-copy RPE65 gene but retain a sufficient number of living retinal cellsThe drug is also the first gene therapy approved by the United States and the European Union to treat hereditary retinal disease (IRD)the structure and function mode of The AGN-151587
AGN-151587 is an AAV5 viral vector loaded with three components, including: two guideRNA (gRNA) gRNA-323 and gRNA-64 controlled by the U6 polymerase III promoter, and Staphylococcus aureus Cas9 expressed by the photoreceptor-specific GRK1 promoterAGN-151587 is injected directly into the patient's photosensitive cells through a retinal injection, delivering a gene editing system to the photosensitive cellsWhen photosensitive cell expression gene editing systems, gRNA-directed gene editing can eliminate or reverse disease-causing IVS26 mutations in the CEP290 gene, thereby improving photosensitive cell functionBecause Cas9 is controlled by a light receptor-specific GRK1 promoter, the system only performs gene editing in photosensitive cells, minimizing potential side effectsBRILLIAN, a multicenter, open label study, will assess the safety, tolerance and efficacy of approximately 18 patients treated at AGN-151587In the study, up to five groups of patients were treated with Three doselevels of AGN-151587The patients in the group included children (3-17 years old) and adult LCA10 patients with genetic screening for pure and compound hetero-mutations involving CEP290 gene 26 ineiko c.2991-1655A-G, and the optimal corrective vision for photosensitive examination to 0.4logMARIn the study, patients were given a single dose of AGN-151587 by a single dose of the eye under the retina after a glass excisionCurrently, some clinical centers in the United States are already recruiting patients for the study, and the first case is expected to take place in the second half of 2019Editas Medicine pipeline assetsLeiber Congenital Black Mandy Disease (LCA) is a group of hereditary retinal degenerative diseases caused by mutations in at least 18 different genesThe disease is the most common cause of hereditary blindness in children, occurring in 2-3 out of every 100,000 live births worldwidePatients develop LCA symptoms in the first few years of life, leading to severe vision loss and potential blindnessThe most common form of disease, LCA10, is a single gene disease caused by a mutation in the CEP290 gene, accounting for about 20%-30% of all LCA patientsMarch 2017, Editas signed a strategic joint and option agreement with Eljian, under which Itas was granted exclusive access to the five genome editing projects for eye diseases, covering the first eye projects for a wide range of eye diseases that are seriously threatened by Editas's two CRISPR genome editing platforms (CRISPR/CasR9 and CRISPR/Cpf1)In August 2018, Aer Jian exercised its option to develop and commercialize AGN-151587 worldwidereference source:1, Editas, Allergan first launch in vivo study of CRISPR-al-ling medicine2, Allergan and Editas Medicine Initiate the Brilliance Phase 1/2 Clinical Of AGN-151587 (EDIT-101) for the Treatment of LCA10original title: Genome Editing in Humans! El Jian/Editas Start Ophthalmology CRISPR Therapy Phase I/II Clinical