Precision BioSciences (DTIL) Guggenheim Securities Emerging Outlook: Biotech Summit 2026 summary

Company overview and technology focus

• Specializes in in vivo gene editing using proprietary ARCUS technology, with 20 years of expertise and a focus on two main programs: chronic hepatitis B and Duchenne muscular dystrophy.

• ARCUS enables precise gene edits, targeting diseases with significant unmet needs and offering solutions where other technologies face limitations.

Duchenne muscular dystrophy (DMD) program

• PBGENE-DMD uses ARCUS to excise exons 45-55, producing a near full-length dystrophin protein, aiming for broader applicability and improved function over micro-dystrophin therapies.

• Preclinical data show 10-15% dystrophin in heart and up to 25% in skeletal muscle, exceeding the 5% threshold linked to clinical benefit.

• The IND for PBGENE-DMD was recently cleared, with plans to dose ambulatory patients in a single-dose, non-escalating phase I trial focused on safety.

• Manufacturing emphasizes high full-to-empty capsid ratios (>85%) to enhance safety and tolerability, with robust oversight of CDMO partners.

• Aggressive immunosuppression and comprehensive supportive care are planned to maximize safety in pediatric patients.

Clinical trial timelines and data expectations

• Initial DMD trial will treat 3-5 patients in 2024, with early safety and dystrophin expression data expected by year-end.

• Early biopsies will provide dystrophin data, with staggered dosing to ensure patient safety.