Precision BioSciences (DTIL) 24th Annual Needham Virtual Healthcare Conference summary

Strategic focus and platform differentiation

• Transitioned to a fully in vivo gene editing company, divesting agricultural and ex vivo CAR T businesses to fund clinical pipeline.

• ARCUS platform is proprietary, highly differentiated, and not derived from bacterial enzymes, offering single-protein simplicity and small size for efficient delivery.

• Over 65 patents protect ARCUS, enabling unique gene editing capabilities, especially for insertion and elimination programs.

• ARCUS enables homology-directed repair, high efficiency insertion, and is advantageous for liver-targeted therapies.

• Focused on applying technology where it can dramatically improve standard of care and achieve functional cures.

Clinical pipeline and program updates

• Lead program PBGene-HBV targets chronic hepatitis B, aiming to eliminate cccDNA and inactivate integrated HBV DNA for functional cure.

• ELIMINATE-B trial is a global phase I/IIA study, enrolling up to 45 patients across five countries, with recent regulatory clearances in the US and UK.

• Initial clinical data showed PBGene-HBV was well tolerated and led to substantial S antigen reductions in two of three patients at the lowest dose.

• Second wholly owned program, PBGene-3243, targets mitochondrial disease, leveraging ARCUS’s ability to cross mitochondrial membranes.

• DMD program (PBGene-DMD) for Duchenne muscular dystrophy is under assessment, aiming for permanent gene correction in up to 60% of patients.

Preclinical and clinical data insights

• Preclinical studies in non-human primates showed high safety and efficacy, with transient, mild transaminase elevations and up to 99% editing after two doses.

• Clinical trial design allows for multiple ascending doses and rapid escalation, optimizing for both safety and efficacy.

• Functional cure is defined as undetectable HBV DNA and S antigen off treatment for at least six months; current standard of care achieves this in only 1–3% of patients.

• Biomarker-driven endpoints and robust monitoring are central to the ELIMINATE-B study.

• Ongoing data sharing expected throughout 2025 as more patients are dosed and cohorts completed.