Zolgensma (onasemnogene abeparvovec-xioi) - Novartis
P5 promoter-mediated incorporation explains REP/CAP manufacturing contaminants in patient liver after rAAV gene therapy. (PubMed, medRxiv) - Sep 25, 2026 - "Together, these findings identify a defined, avoidable mechanism by which REP/CAP manufacturing contaminants arise. Positioning of the P5 promoter within the manufacturing plasmid is therefore a modifiable determinant of rAAV product purity and the transfer of rAAV DNA contaminants to recipient patients." 
Journal • Gene Therapies • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases
http://www.ncbi.nlm.nih.gov/pubmed/42779866
 
Jeremy Chase Crawford; Judith Breuer; Li-An K Brown; Mark A Brimble; Sarah Buddle; Shaoyuan Tan
 
Sep 25, 2026
 
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