Precision BioSciences to present preclinical PBGENE-HBV data

Precision BioSciences (DTIL) will present preclinical data supporting the advancement of PBGENE-HBV into clinical development. The company will also outline the design of its first-in-human study, ELIMINATE-B, in patients with chronic hepatitis B. Company management will present these updates and host panel discussions on the unmet need in chronic hepatitis B and the approach to treatment with PBGENE-HBV during a virtual event being held November 15 before commencement of the American Association for the Study of Liver Diseases, or AASLD, meeting. Precision will present preclinical data generated to date, which support the progression of PBGENE-HBV into a first-in-human clinical trial. The company will share safety, tolerability, and efficacy signals observed through an array of preclinical models. Comprehensive off-target analysis demonstrated a high degree of specificity for PBGENE-HBV, with no increased risks of translocations or integrations in HBV-infected human liver cells; PBGENE-HBV was well tolerated over multiple administrations in mice and non-human primates (NHPs), with rapid clearance after each dose administration, transient transaminase elevations which resolved rapidly, and non-adverse changes in blood parameters; PBGENE-HBV does not distribute to germ cells, as evidenced by NHP studies and PBGENE-HBV’s high-quality mRNA and optimized LNP formulation contributed to a compelling safety profile. PBGENE-HBV effectively distributed to all hepatocytes in the liver; PBGENE-HBV demonstrated 99% viral DNA editing in NHPs; Confirmed PBGENE-HBV’s dual mechanism with elimination of cccDNA observed in primary human hepatocyte, mouse, and NHP models and inactivation of integrated HBV DNA observed in transgenic mouse models and HBV cell lines and observed sustained declines in key viral biomarkers, HBV DNA and hepatitis B surface antigen, indicative of a functional cure in transgenic mouse models following administration of PBGENE-HBV and nucleoside analogue withdrawal. Based on these data, Precision has submitted clinical trial applications to authorities in multiple geographies and has so far received clearance to initiate its Phase 1 study in Moldova. Additional regulatory applications are under review or planned for other jurisdictions, including a U.S. IND.