Precision BioSciences (DTIL) Sidoti May Micro-Cap Virtual Conference summary

Key program updates

• Advanced to clinical stage with PBGENE-HBV for hepatitis B, now treating patients at three sites across five countries, with data readouts expected in 2025.

• Announced PBGENE-DMD for Duchenne muscular dystrophy, targeting 60% of patients with a novel gene editing approach; IND/CTA filing planned for later in 2025 and data expected in 2026.

• Partnership with Eacure on an OTC deficiency program has shown a complete response in the first treated infant, validating ARCUS technology.

• All three programs are expected to reach phase I data readouts within the current cash runway, which extends into the second half of 2026.

• Received FDA fast-track designation for the hepatitis B program in March 2025.

ARCUS gene editing platform highlights

• ARCUS is a proprietary, non-CRISPR gene editing tool with over 65 patents, offering unique advantages in cut type, size, and simplicity.

• Its small size allows delivery via both lipid nanoparticles and AAV, enabling access to a range of tissues and the ability to fit two nucleases in a single AAV.

• ARCUS operates as a single protein, eliminating the need for guide RNAs and reducing complexity compared to CRISPR.

• Demonstrated ability to efficiently target and edit disease-causing DNA, including in liver and muscle tissues.

• Safety is prioritized through protein engineering to minimize off-target effects, with no serious adverse events reported in clinical trials to date.

Clinical and preclinical data insights

• PBGENE-HBV has shown substantial antiviral activity and a strong safety profile at initial dose levels, with no serious adverse events.

• The Eliminate B trial uses a multi-dose, ascending design to maximize efficacy and safety, with early signs of efficacy already observed.

• PBGENE-DMD preclinical studies in mice demonstrated functional muscle improvement over time, surpassing current therapies that only stabilize or slow decline.

• The DMD program targets the root genetic cause, aiming for long-term durability by editing muscle stem cells and restoring near full-length dystrophin protein.

• The dystrophin protein produced mimics that found in milder Becker dystrophy patients, who often live into their 60s and 70s.