DTIL — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-05-05

• PBGENE-HBV is our wholly owned in vivo gene editing program under investigation in a global first-in-human clinical trial, ELIMINATE-B, which is designed to be a potentially curative treatment for chronic hepatitis B infection. In patients with chronic hepatitis B, cccDNA acts as the template to mak…

• As part of the ongoing assessment of the safety and efficacy profile of PBGENE-HBV after repeat doses in Part 1 dose finding, we have treated 16 patients with 38 administrations of PBGENE-HBV across five cohorts evaluating the impact of escalating dose levels as well as 8-week and 4-week dosing inte…

• Looking ahead, we expect to share further clinical data from the PBGENE-HBV program at hepatitis-focused medical conferences throughout 2026, starting with the European Association for the Study of the Liver (“EASL”).

• In April 2026, we announced that a late-breaking poster for PBGENE-HBV was accepted for presentation at the EASL Congress 2026. The poster titled, “First evidence of elimination and inactivation of cccDNA in liver biopsies collected from patients with chronic hepatitis B treated with PBGENE-HBV” wil…

• Also in April 2026, we announced that we received Clinical Trial Application (“CTA”) approval to expand ELIMINATE-B into France and Romania, broadening the study’s footprint in Europe. Site initiation activities are underway and initial patient screening in those countries is expected in the second …

Text removed vs the prior filing · source: 10-Q · 2025-11-03

• We are leveraging our proprietary ARCUS genome editing platform to advance in vivo gene editing programs that go beyond gene knockouts in the liver and carry out more sophisticated edits such as gene insertions, gene excision, and gene elimination, unlocking a broader potential for ARCUS in vivo gen…

• PBGENE-HBV is our wholly owned in vivo gene editing program under investigation in a global first-in-human clinical trial, which is designed to be a potentially curative treatment for chronic Hepatitis B infection. PBGENE-HBV is the first and only potentially curative gene editing program to enter t…

• In August 2025, we announced Phase 1 safety and efficacy data for Cohort 1 (0.2 mg/kg) and initial safety data from Cohort 2 (0.4 mg/kg) in the ELIMINATE-B study and subsequently presented the data in September 2025 at the 6th International Coalition to Eliminate HBV Cure Symposium. Following multip…

• We have been selected to deliver a late-breaking oral presentation at the upcoming Liver Meeting 2025 during the 75th American Association for the Study of Liver Diseases on November 10, 2025. The oral presentation will feature new data from the first two cohorts of the ongoing Phase 1 ELIMINATE-B T…

• Duchenne muscular dystrophy, or DMD, is a genetic disease caused by mutations in the dystrophin gene that prevent production of the dystrophin protein and affects approximately 15,000 patients in the United States alone. There are currently no approved therapies with curative intent that can drive d…

Text added vs the prior filing · source: 10-Q · 2026-05-05

• Many of our current or potential competitors, either alone or with their collaborators, have significantly greater financial resources and expertise in research and development, manufacturing, preclinical testing, conducting clinical trials, obtaining regulatory approvals and marketing approved prod…

• We face an inherent risk of product liability and professional indemnity exposure related to the testing in clinical trials of our product candidates. We will face an even greater liability risk if we commercially sell any products that we or our

• collaborators may develop for human use. Manufacturing defects, errors in product distribution or storage processes, improper administration or application and known or unknown side effects of product usage may result in liability claims against us or third parties with which we have relationships. …

• Regulatory requirements governing products created with genome editing technology or involving gene therapy treatment have changed frequently and will likely continue to change in the future. Approvals by one regulatory agency may not be indicative of what any other regulatory agency may require for…

• CBER on its review. Our product candidates will need to meet safety, purity, and potency standards applicable to any new biologic under the regulatory framework administered by the FDA.

Text removed vs the prior filing · source: 10-Q · 2025-11-03

• Many of our current or potential competitors, either alone or with their collaborators, have significantly greater financial resources and expertise in research and development, manufacturing, preclinical testing, conducting clinical trials,

• We face an inherent risk of product liability and professional indemnity exposure related to the testing in clinical trials of our product candidates. We will face an even greater liability risk if we commercially sell any products that we or our collaborators may develop for human use. Manufacturin…

• Regulatory requirements governing products created with genome editing technology or involving gene therapy treatment have changed frequently and will likely continue to change in the future. Approvals by one regulatory agency may not be

• indicative of what any other regulatory agency may require for approval, and there has historically been substantial, and sometimes uncoordinated, overlap in those responsible for regulation of gene therapy products, cell therapy products and other products created with genome editing technology. Fo…

• patients who received treatment with BCMA- or CD19-directed autologous CAR T cell immunotherapies, and in January 2024, the FDA required the manufacturers of certain CAR-T therapies to add boxed warnings to product labeling cautioning against the risk of T-cell malignancies. Although we are no longe…