Precision BioSciences to Present Preclinical Gene Editing Data on its ARCUS-Based Chronic Hepatitis B Program at the HEP DART 2021 Conference
Abstract Number and Title: Abstract #03. Targeting Hepatitis B cccDNA with a Sequence-Specific ARCUS Nuclease to Eliminate Hepatitis B Virus In
Abstracts for the HEP DART 2021 Meeting are available on the conference web site, at https://static1.squarespace.com/static/5c912d7af8135a45d18dd805/t/61aa80a73f24a36f20f71eca/1638564010806/hepdart-2021-abstract-book-final%5B26%5D.pdf.
“Current standard-of-care treatments for chronic hepatitis B work by lowering or suppressing the amount of hepatitis B virus found in infected cells in the body, rarely clearing the virus fully and leaving behind a DNA template for HBV to replicate,” said
In this preclinical study, ARCUS efficiently targeted and degraded HBV cccDNA and reduced expression of HBV s-antigen (HBsAg) by 77% in HBV-infected primary human hepatocytes. To evaluate ARCUS in vivo, mouse and non-human primate models were developed that utilized an episomal adeno-associated virus (AAV) containing a portion of the HBV genome to serve as a surrogate for cccDNA. In both episomal models, a robust decrease in AAV copy number and high on-target editing in remaining AAV was observed, and a durable 96% reduction of HBsAg was further observed in mice.
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