Precision BioSciences (DTIL) 25th Annual Needham Virtual Healthcare Conference summary

Platform and technology overview

• ARCUS gene editing platform, developed from a homing endonuclease in green algae, enables precise gene editing across various tissues and diseases.

• ARCUS is differentiated by its unique DNA cut, small size for flexible delivery, and single-component simplicity, allowing efficient editing without guide RNA.

• Over 75 patents have been issued for ARCUS, supporting a robust intellectual property position.

• ARCUS enables both in vivo and ex vivo applications, with successful preclinical and early clinical results.

Pipeline programs and clinical progress

• Two main in vivo programs: PBGENE-HBV for chronic hepatitis B and PBGENE-DMD for Duchenne Muscular Dystrophy, both addressing large unmet needs.

• PBGENE-HBV is in a Phase 1/2 trial (ELIMINATE-B), with additional data expected in 2026; early results show dose-dependent, durable S antigen declines and proof of gene editing in liver biopsies.

• PBGENE-DMD targets up to 60% of DMD patients, aiming for a single administration therapy producing near full-length dystrophin; IND clearance received, with first patient dosing planned in the first half of the year.

• Preclinical DMD data show sustained dystrophin expression and functional improvement in mice, with protein levels exceeding therapeutic thresholds.

Strategic partnerships and financial outlook

• Partnerships with iECURE (OTC deficiency), Imugene (oncology), and TG Therapeutics (autoimmune) have generated milestone payments, supporting ongoing trials.

• iECURE's ARCUS-based program achieved a complete clinical response in OTC deficiency, demonstrating technical feasibility.

• Cash position of $137 million at year-end, expected to fund operations through 2028.

• Multiple data events anticipated in 2026 and 2027 for both lead programs.