AGTC - AGTC reports process development advances of XLRP gene therapy candidate at ASGCT

Applied Genetic Technologies (AGTC) presents new data from two studies related to improvements in the manufacturing process for rAAV2tYF-GRK1-RPGRco, a gene therapy candidate for the treatment of X-linked retinitis pigmentosa ((XLRP)). Subretinal Rd9 Mouse Study to Compare RPGR Expression Pre- and Post-Manufacturing Process Improvements:This abstract describes a study designed to compare transgene expression between vectors produced with the original and improved processes.Study results show that these changes resulted in an improved full/empty capsid ratio from 30% to greater than 80%, reduced process residuals (Host cell DNA and Protein), and an improved ratio of infectious particles (< 4.0 total particles/infectious particle).Immunohistochemistry staining of RPGR expression was scored as equivalent in retinal area (most showed >50% photoreceptor positivity) and intensity of staining following subretinal injection of XLRP vectors produced under each process into the eyes of Rd9 mutant mice, an animal model for XLRP due to mutations in the RPGR gene.These findings

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AGTC reports process development advances of XLRP gene therapy candidate at ASGCT