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    Home > Active Ingredient News > Drugs Articles > Eye gene editing therapy agn-151587 (edit-101) officially launched phase I / II clinical research

    Eye gene editing therapy agn-151587 (edit-101) officially launched phase I / II clinical research

    • Last Update: 2019-07-26
    • Source: Internet
    • Author: User
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    Leber's congenital amaurosis (LCA) is a group of inherited retinal degenerative diseases caused by mutations of at least 18 different genes The disease is the most common cause of hereditary blindness in children, occurring in 2-3 of every 100000 live births worldwide Recently, eierjian and the genome editing company (http:// editas medicine jointly announced Brilliance (nct03872479), a phase I / II clinical study of its eye gene editing therapy agn-151587 (edit-101), has started the enrollment of patients About agn-151587 Agn-151587 is being developed for the treatment of Leber's congenital amaurosis 10, which is a genetic blindness caused by the mutation of centrosomal protein 290 (CEP290) Agn-151587 is an AAV5 virus vector loaded with three components, including two guide RNA (gRNA) grna-323 and grna-64 controlled by the U6 polymerase III promoter, and Staphylococcus aureus cas9 expressed through the GRK1 promoter specific to the photoreceptor Agn-151587 is injected directly into the patient's photoreceptor cells by subretinal injection, and the gene editing system is delivered to the photoreceptor cells When photoreceptors express gene editing system, gRNA guided gene editing can eliminate or reverse the pathogenic ivs26 mutation in CEP290 gene, thus improving the function of photoreceptors Because cas9 is controlled by the photoreceptor specific GRK1 promoter, gene editing occurs only in photoreceptor cells, thus minimizing potential side effects About brilliance brilliance is a multicenter, open label study that will evaluate the safety, tolerability, and efficacy of agn-151587 in approximately 18 patients In the study, up to five groups of patients will receive three doses of agn-151587 The patients included children (3-17 years old) and adult lca10 patients with various vision ranges These patients had homozygous or compound heterozygous mutations involving c.2991 + 1655a > G in intron 26 of CEP290 gene through gene screening, and the best corrected visual acuity was 0.4logmar on photosensitive examination In the study, a single dose of agn-151587 was administered to one eye after vitrectomy At present, some clinical centers in the United States are already recruiting patients for this study, and the first patient treatment is expected in the second half of 2019  
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