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Khabre desk
Recent reporting, analysis and explainers from the gene therapy desk.
science
A bioRxiv preprint reports that neonatal AAV-SIL1 gene therapy prevented neurological and muscle disease in a mouse model of Marinesco-Sjögren syndrome. The treatment preserved cerebellar Purkinje cells and maintained its effects through 26 weeks.
health
A multicentre phase 1 trial in China tested the AAV gene therapy BBM-H901 in 11 adolescents with severe or moderately severe hemophilia B. At 52 weeks, mean factor IX activity was 41.8 IU/dl and the mean annualized bleeding rate had fallen from 13.9 to 0.5.
health
A medRxiv preprint identifies a possible manufacturing mechanism behind REP/CAP DNA sequences found in the liver of a patient treated with the rAAV gene therapy Zolgensma. The proposed mechanism involves the placement of the AAV P5 promoter in a manufacturing plasmid.