Precision BioSciences Presents Preclinical Data Highlighting Capabilities of ARCUS for Gene Insertion and Excision at the European Society of Gene & Cell Therapy (ESGCT) 30th Annual Congress
- ARCUS showed durable, high-efficiency gene insertion capabilities in NHPs and comparison to CRISPR-Cas9
- Data demonstrates ability of ARCUS to achieve large gene excisions enabling significant functional muscle improvement in a DMD mouse model
“We believe ARCUS has the potential to redefine gene editing by enabling sophisticated gene edits such as gene insertion, excision, and elimination,” said
Title: Unique features of ARCUS nucleases enable high efficiency, targeted gene insertion in vivo
Poster Number: #P641
Date and Time:
Location: Gare Maritime
In preclinical work presented today, ARCUS showed high efficiency gene insertion in vitro (primary human T cells and hepatocytes) and in vivo (infant and adult NHPs). ARCUS’ 3’ overhangs were shown to drive high efficiency gene insertion compared to blunt cuts, and in vivo ARCUS demonstrated high efficiency and durable insertion in newborn and infant NHPs when used with OTC and F9 transgene insertion templates after adeno-associated virus (AAV) delivery. ARCUS showed up to ~45% insertion when administered by LNP along with an AAV carrying a gene insertion template in adult NHPs, and demonstrated high efficiency gene insertion via HDR in nondividing, primary human hepatocytes.
Title: ARCUS-mediated excision of the “hot spot” region of the human dystrophin gene results in functional improvement in a mouse model of Duchenne muscular dystrophy (DMD)
Poster Number: Poster #P653
Date and Time:
Location: Gare Maritime
In the data on display today using early generation ARCUS nucleases, Precision scientists observed the edited dystrophin protein variant in multiple tissue types frequently involved in progression of DMD, including heart, diaphragm, and skeletal muscle. Furthermore, the maximum force output of the gastrocnemius muscle in ARCUS-treated animals was significantly improved compared to untreated mice, reaching 86% of the maximum force output levels observed in non-diseased, control animals.
About Duchenne muscular dystrophy
DMD is a genetic disorder associated with mutations in the dystrophin gene that prevent production of the dystrophin protein. Dystrophin stabilizes the cell membrane during muscle contraction to prevent damage, and the absence of intact dystrophin protein leads to inflammation, fibrosis, and progressive loss of muscle function and mass. Over time, children with DMD will develop problems walking and breathing, eventually leading to death in the second or third decade of life due to progressive cardiomyopathy and respiratory insufficiency. DMD occurs in 1 in 3,500 to 5,000 male births, and currently there are limited approved therapies available for patients.
ARCUS is a proprietary genome editing technology discovered and developed by scientists at
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding expected conference participation and disclosure of preclinical data, the ability of ARCUS to make predictable, highly consistent, therapeutic edits as well as durable, high-efficiency gene insertion capabilities through HDR, including NHPs, the potential to redefine gene editing by enabling sophisticated gene edits such as gene insertion, excision, and elimination, the clinical development, nomination, and goals of our PBGENE-DMD program, therapeutic potential of an ARCUS gene editing approach for the treatment of DMD, including ability of ARCUS to achieve large gene excisions, enabling significant functional muscle improvement in a DMD mouse model, and expected safety, efficacy, and benefit of our gene editing approaches. In some cases, you can identify forward-looking statements by terms such as “aim,” “anticipate,” “approach,” “believe,” “contemplate,” “could,” “estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,” “plan,” “possible,” “potential,” “predict,” “project,” “pursue,” “should,” “target,” “will,” “would,” or the negative thereof and similar words and expressions.
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