Precision BioSciences (DTIL) H.C. Wainwright Liver Disease Virtual Conference summary

Conference overview

• Combined viral hepatitis and MASH conferences to broaden focus to liver cancer and innovative biotech solutions.

• Featured presentations from leading biotech companies and key opinion leaders in hepatitis and liver disease.

• Highlighted ARCUS gene editing platform for in vivo therapies targeting genetic and infectious liver diseases.

ARCUS platform and differentiation

• ARCUS is a proprietary, compact gene editing nuclease derived from green algae, engineered for high specificity.

• Unique features include a four-base pair overhang cut, small size (~1,000 bases), and single-protein simplicity.

• Small size enables flexible delivery via LNP and AAV technologies, improving mRNA quality and safety.

• Single-protein design contrasts with multi-component CRISPR systems, potentially increasing efficiency.

Rationale for targeting HBV

• Chosen due to high unmet need; current functional cure rates for HBV are only 1%-2% with standard care.

• ARCUS’s small size and simplicity are advantageous for targeting cccDNA in HBV using LNP delivery.

• Goal is to achieve a complete cure by eliminating cccDNA and inactivating integrated HBV DNA, not just functional cure.