Precision BioSciences publishes research on ARCUS gene editing platform

DURHAM, N.C. - Precision BioSciences (NASDAQ:DTIL), a clinical-stage biotechnology company with a market capitalization of $67 million and strong liquidity position, announced the publication of research demonstrating the capabilities of its ARCUS gene editing platform in the journal Nucleic Acids Research. The study highlights the platform’s ability to achieve high-efficiency gene insertions and various DNA edits.

The research shows ARCUS nucleases can achieve transgene insertion rates exceeding 85% in T lymphocytes and up to 40% in non-dividing primary human hepatocytes. According to the publication, the platform can facilitate gene insertion, single base editing, specific deletions, and replacement of large stretches of genomic DNA. The company’s stock has shown strong momentum, with a 25% gain over the past six months, though InvestingPro analysis indicates significant volatility in share price movements.

"ARCUS continues to demonstrate capabilities beyond what is possible with other current genome editing technologies," said Jeff Smith, Co-Founder and Chief Research Officer at Precision BioSciences, in the press release. While the company maintains a healthy current ratio of 5.18 and holds more cash than debt, InvestingPro data reveals rapid cash consumption that investors should monitor. Get access to 10+ additional exclusive ProTips and comprehensive financial metrics with an InvestingPro subscription.

The company stated that a key differentiating feature of ARCUS is its ability to generate 3’ overhang ends at DNA break sites, which facilitates gene insertion in both dividing and non-dividing cells.

Precision BioSciences is developing several in vivo gene editing programs using the ARCUS platform, including ECUR-506 for gene insertion, PBGENE-HBV for gene elimination, and PBGENE-DMD for excision of large DNA sequences.

The clinical stage gene editing company is focused on using its proprietary platform to develop treatments for genetic and infectious diseases where limited treatment options exist.

The information in this article is based on a press release statement from Precision BioSciences.

In other recent news, Precision BioSciences has reported positive data from its Phase 1 ELIMINATE-B trial for the PBGENE-HBV gene editing therapy aimed at treating chronic Hepatitis B. The trial demonstrated that the therapy was well-tolerated and showed significant activity in patients, with promising early results indicating substantial viral reductions in the lowest dose cohort. Additionally, the company announced it will receive a U.S. patent for this gene editing therapy, extending protection until March 2042. This patent covers the ARCUS nuclease used in targeting HBV DNA, adding to similar patents already granted in Europe and Hong Kong.

Precision BioSciences also updated executive contracts, modifying severance terms for key officers, including the CEO and CFO. In another development, the U.S. FDA granted Orphan Drug Designation to the company’s PBGENE-DMD treatment for Duchenne muscular dystrophy, a rare genetic disorder. These recent developments highlight the company’s ongoing efforts in advancing its gene editing therapies and securing intellectual property rights.

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