Precision BioSciences Inc（DTIL）のニュースとイベントDTILPrecision BioSciences Inc

Precision BioSciences Reports PBGENE-HBV Clinical Data, 100% Patients Achieve pgRNA Loss

Precision BioSciences announced new and updated Phase 1 clinical data from the ongoing ELIMINATE-B study of PBGENE-HBV to be presented in a late-breaking oral presentation on Sunday, November 8 at 2:15pm MST, at the American Association for the Study of Liver Diseases The Liver Meeting 2026. PBGENE-HBV is the first and only in vivo gene editing therapy designed to eliminate the root cause of chronic hepatitis B, covalently closed circular DNA, to drive viral cure measured by sustained undetectable HBV DNA. The oral presentation will be delivered by our ELIMINATE-B lead investigator, Man Fung Yuen, MD, Ph.D., Chair Professor of The University of Hong Kong and the Chief of the Division of Gastroenterology and Hepatology, Queen Mary Hospital. The presentation features new and updated data from 25 patients across 59 administrations in the Phase 1 ELIMINATE-B trial.As of the data cutoff date, September 28, 2026, 25 patients had received a total of 59 administrations of PBGENE-HBV across four dose levels. "We're thrilled to share the complete and durable loss of pgRNA in 100% of evaluable patients at our go-forward dose of 0.4 mg/kg, as well as durable, ongoing pgRNA loss in all patients who had at least 6-months of follow up, which we believe is indicative of permanent elimination of cccDNA in the liver. In total, across all dose levels, 11 evaluable patients achieved pgRNA loss to undetectable and remained undetectable through data cut off. The highlight of the data was demonstrating pgRNA loss to undetectable levels in our first HBeAg positive patient dosed on study, indicating potent effect of PBGENE-HBV in a patient population that is very difficult to treat due to logarithmically higher amounts of cccDNA and viral load in the liver," said Cassie Gorsuch, PhD, Chief Scientific Officer of Precision BioSciences. Among 12 evaluable patients with detectable pregenomic RNA at baseline, 11 achieved undetectable pgRNA following treatment with PBGENE-HBV, including all six evaluable patients at 0.4 mg/kg. The remaining patient, dosed with only two administrations at 0.65 mg/kg, experienced a 95% reduction in pgRNA at the time of the data cutoff and will continue to be followed to evaluate pgRNA at go forward visits. In groundbreaking data, the first HBeAg-positive patient treated in the study achieved undetectable pgRNA upon the second administration of PBGENE-HBV at 0.4 mg/kg, declining from 5,750 copies/mL at baseline. As a blood-based biomarker of cccDNA transcription, pgRNA provides evidence of PBGENE-HBV's intended mechanism of cccDNA elimination in the liver. Among patients who achieved undetectable pgRNA and had at least six months of follow-up, 100% maintained undetectable pgRNA through the data cutoff. The longest observed duration of pgRNA loss now approaches one year, extending the findings reported in May 2026. The sustained absence of detectable pgRNA across patients provides validation evidence and confidence that as designed, PBGENE-HBV is eliminating the cccDNA viral reservoir that drives chronic hepatitis B. Paired liver biopsies from two patients treated with PBGENE-HBV at 0.4 mg/kg, the go forward Phase 2 dose demonstrated approximately 1-log (10-fold) and 1.5-log reductions in cccDNA , respectively across multiple patients. These findings reproduce and extend the initial liver biopsy evidence of cccDNA elimination reported previously at EASL 2026. The concordance between elimination of cccDNA-derived transcripts in liver tissue and loss of circulating pgRNA provides additional evidence supporting PBGENE-HBV's intended mechanism of directly targeting and eliminating the persistent cccDNA viral reservoir responsible for chronic hepatitis B infection. All 24 evaluable patients experienced reductions in the secondary biomarker hepatitis B surface antigen (HBsAg) following treatment with PBGENE-HBV at all dose levels. Maximal HBsAg reductions approached 94% at 0.4 mg/kg, with similar antiviral activity observed in patients with and without detectable pgRNA at baseline. HBsAg is produced from both cccDNA and integrated HBV DNA, therefore elimination of cccDNA is expected to result in sustained and durable reduction of HBsAg. The HBsAg findings provide consistent supportive evidence of antiviral activity across a broad patient population and, together with pgRNA loss and liver biopsy findings, further support PBGENE-HBV's intended mechanism of action and the expected go-forward dose. PBGENE-HBV demonstrated a well-characterized and manageable safety profile across 59 administrations in 25 patients, with no dose-limiting toxicities observed. The most common adverse events were infusion-related reactions consistent with lipid nanoparticle effects, with onset and resolution generally occurring within 24 hours of infusion. The 0.4mg/kg dose was better tolerated than the 0.65mg/kg, dose, allowing all dose administrations to be delivered and further supporting the go-forward dose in Phase 2. At the expected Phase 2 dose of 0.4 mg/kg, no serious adverse events have been observed. Under the current infusion protocol designed to mitigate potential LNP-related inflammatory responses, no Grade 3 or higher hypotension events have occurred across 15 administrations in eight patients at the Phase 2 dose of 0.4 mg/kg. Transient, reversible elevations in alanine aminotransferase and aspartate aminotransferase have been observed following PBGENE-HBV administration, consistent with LNP-associated effects. These laboratory abnormalities were asymptomatic, with no associated elevations in bilirubin, cases of Hy's Law or evidence of hepatic synthetic dysfunction across any dose level. At 0.4 mg/kg under the current infusion protocol, one delayed ALT/AST elevation was observed following the third administration, with peak ALT of 306 U/L and AST of 278 U/L. The elevation was transient and resolved to normal with no intervention required and no associated bilirubin changes were observed. Collectively, the antiviral activity and safety profile of PBGENE-HBV support selection of 0.4 mg/kg for further evaluation in Phase 2.

Precision BioSciences Prices 10.23M Share Offering, Raising Approximately $125 Million

Precision BioSciences announced the pricing of an underwritten offering of 10,235,077 shares of its common stock at a price of $7.75 per share and, in lieu of common stock to certain investors, pre-funded warrants to purchase up to 5,893,964 shares of its common stock at a price of $7.749995 per pre-funded warrant, which represents the per share offering price for the shares of common stock less the $0.000005 per share exercise price for each pre-funded warrant. The gross proceeds to Precision from the offering, before deducting the underwriting discounts and commissions and offering expenses, are expected to be approximately $125 million. The deal includes participation from new and existing investors, including First Light Asset Management, MPM BioImpact, Bleichroeder LP, Lynx1 Capital Management, Readout Capital, Aberdeen Investments, Driehaus Capital Management, as well as other leading life sciences investors. The offering is expected to close on or about November 6, 2026, subject to customary closing conditions. All shares of common stock and pre-funded warrants to be sold in the offering will be sold by Precision. Precision intends to use the net proceeds of the offering to fund the continued research and development of PBGENE-HBV and PBGENE-DMD, and for working capital and general corporate purposes. TD Cowen and Guggenheim Securities are acting as joint book-running managers for the offering.

Precision BioSciences Completes Safety Review of PBGENE-DMD

Precision BioSciences announced that the independent Data Safety Monitoring Board has completed its review of safety data from the first patient treated with PBGENE-DMD in the Phase 1/2 FUNCTION-DMD clinical trial for Duchenne muscular dystrophy. No serious adverse events or adverse events of special interest were reported in the first patient through week eight. Following completion of the patient's initial eight-week safety observation period, the DSMB identified no safety concerns and endorsed proceeding with dosing additional patients in Part 1 of the study.