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December 7th, 2020 - December 7, 2020 – Want to learn more about NxGEN CpG-depletion technology? View our brochure below.

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November 19, 2020 – You’ve probably seen the plethora of papers and presentations in 2020 demonstrating that stable transgene expression in human clinical trials is dependent on the AAV vector genome CpG motifs. This confirms the preclinical studies performed nearly 8 years ago by Dr. Susan Faust and Dr. Joseph Rabinowitz–both members of NxGEN Vector Solutions–and pioneers of CpG-depleted AAV vectors (NxGEN Technology). The desirability of CpG depletion of AAV vectors for the purpose of reducing the immune response to the vector and establishing long-term transgene expression is very hot in the manufacturing sector and of great interest to gene therapists.
February 14, 2021—An editorial published in Blood by Lindsey A. George, M.D. (Children’s Hospital of Philadelphia) entitled No CpGs for AAVs? comments on the human clinical trial, NCT01687608, which explored a self-complementing optimized AAV vector to deliver the human FIX-Padua gene (Konkle et al., 2019). The treatment, BAX335, also known as AskBio009, employed an increased number of CpG di-nucleotides (ie. from 19 to 99) in the FIX open reading frame. Ultimately, the results of the clinical trial confirmed that long-term transgene expression was not achieved with AAV vectors having increased CpG content in the coding sequence for the transgene despite steroid intervention due to the innate immune stimulatory effect of CpG motifs enriched within their vector cassette.

NxGEN Vector Solutions, LLC
455 Massachusetts Ave., N.W., Suite 369
Washington, D.C. 20001
p: 202.765.7475
www.nxgenvectorsolutions.com

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