Precision BioSciences (DTIL) Sidoti's Year End Virtual Investor Conference summary

Strategic focus and financial position

• Company is exclusively focused on two in-vivo gene editing programs: PBGENE-HBV for hepatitis B and PBGENE-DMD for Duchenne muscular dystrophy.

• Recent $75 million financing extends cash runway through 2028, supporting clinical milestones and data readouts.

• ARCUS gene editing platform has demonstrated clinical validation in multiple indications, including partnerships and internal programs.

• Ongoing discussions with potential partners for commercialization, especially for the HBV program.

• Company maintains a disciplined, focused approach to capital allocation and program execution.

PBGENE-HBV (Hepatitis B) program progress

• PBGENE-HBV targets the root cause of hepatitis B by eliminating cccDNA, aiming for a functional cure.

• Phase 1 ELIMINATE-B trial has shown dose-dependent antiviral activity and a manageable safety profile across three cohorts.

• Higher dose cohorts demonstrate sustained S antigen reduction, with some patients nearing levels where cure testing is possible.

• Plans to complete cohort three dosing, explore shorter dosing intervals, and expand to more patients before phase 2.

• Data readouts expected at major liver conferences and throughout 2026.

PBGENE-DMD (Duchenne muscular dystrophy) program progress

• PBGENE-DMD uses gene editing to excise mutations in exons 45-55, potentially benefiting up to 60% of DMD patients.

• Preclinical studies show restoration of functional dystrophin protein above the 5% threshold linked to milder disease.

• Function-DMB trial designed as a phase 1/2/3 study, aiming to enroll first patients in 2026 and read out data by end of 2026.

• Safety is prioritized with aggressive immunomodulation and top clinical sites.

• Potential for BLA filing by end of 2028 if data are positive.