Chunjuan Song, Thomas J Conlon, Wen-Tao Deng, Kirsten E Coleman, Ping Zhu, Cayrn Plummer, Savitri Mandapati, Mailin Van Hoosear, Kari B Green, Peter Sonnentag, Alok K Sharma, Adrian Timmers, Paulette Robinson, David R Knop, William W Hauswirth, Jeffrey David Chulay, Mark S Shearman, Guo-Jie Ye
AGTC is developing a recombinant adeno-associated virus (rAAV) vector AGTC-501, also designated AAV2tYF-GRK1-RPGRco, to treat retinitis pigmentosa (RP) in patients with mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. The vector contains a codon-optimized human RPGR cDNA (RPGRco) driven by a photoreceptor-specific promoter (G protein-coupled receptor kinase 1, GRK1) and is packaged in an AAV2 capsid with three surface tyrosine residues changed to phenylalanine (AAV2tYF). We conducted a safety and potency study of this vector administered by subretinal injection in the naturally occurring RPGR-deficient Rd9 mouse model...
October 3, 2018: Human Gene Therapy. Clinical Development