Precision BioSciences

Precision BioSciences has treated the first patient in the Phase 1/2 FUNCTION‑DMD study with PBGENE‑DMD. The therapy uses two ARCUS nucleases delivered in an AAV vector to remove exons 45–55 of the dystrophin gene to produce a near‑functional dystrophin; the approach could apply to up to 60% of DMD patients with mutations in this region. The trial enrolls ambulatory children aged two to seven; the company expects initial safety data by the end of 2026. PBGENE‑DMD has already received FDA orphan‑drug and fast‑track designation and is eligible for a Priority Review Voucher. » Más en de.investing.com

Effective August 1, Alex Kelly will move from CFO into the newly created role of chief operating officer and take over customer-facing areas. Naresh Tanna will become the new chief financial officer and will be responsible for finance, reporting and investor relations. Cindy Atwell remains chief development officer; additionally, Cassie Gorsuch was hired as chief scientific officer, while the company presents clinical data on PBGENE-HBV and PBGENE-DMD and continues to develop its ARCUS gene-editing platform. » Más en de.investing.com

Precision BioSciences jumped in pre-market trading after the company presented data from the ELIMINATE-B study at the EASL congress. Biopsy analyses show that PBGENE-HBV reduced cccDNA-derived transcripts by about 1 log and for the first time demonstrate elimination/inactivation of cccDNA in liver biopsies. The study established pgRNA as a biomarker and reported loss of this pgRNA in all patients who had detectable pgRNA at baseline; there were no dose-limiting toxicities, only transient infusion-related reactions and reversible ALT/AST elevations. Precision expects further study updates through the end of 2026. » Más en de.investing.com