Precision BioSciences (DTIL) investor relations material

Company overview and technology platform

• Focused on developing gene editing therapeutics for diseases with high unmet need, leveraging the proprietary ARCUS platform with over 75 patents and clinical validation in multiple programs.

• ARCUS is derived from a homing endonuclease and is wholly owned, enabling high-efficiency gene editing.

Clinical pipeline and partnered programs

• PBGENE-HBV for chronic hepatitis B has treated 13 patients across 5 cohorts, with ongoing data collection and updates expected in 2026.

• PBGENE-DMD targets Duchenne muscular dystrophy (DMD), aiming for durable muscle improvement in ~60% of patients; initial clinical data expected late 2026.

• Partnered programs include gene insertion for OTC deficiency and CAR T therapies in hematology and autoimmune indications, with recent clinical milestones achieved.

PBGENE-DMD: therapeutic rationale and differentiation

• Designed to excise the 'hot-spot' region (exons 45-55) of the dystrophin gene, aiming for permanent restoration of muscle function.

• Seeks to overcome limitations of current therapies by enabling single administration, broad applicability, and near full-length dystrophin protein expression.

• As little as 5% expression of functional dystrophin is expected to provide clinical benefit, with the approach designed for long-term durability independent of AAV persistence.