Gene Editing
PBGENE-HBV, a First-in-class Gene Editing Therapy for Chronic Hepatitis B, Demonstrates Safety and Antiviral Activity in Early Cohorts
Yuen, MF., et.al. (2025) American Association for the Study of Liver Diseases.
Initial safety data from ELIMINATE-B, the first clinical trial of a gene editing treatment for chronic hepatitis B
Sulkowski, Mark, et. al. (2025) American Society of Gene & Cell Therapy
Systemic Delivery of a Mitochondria-Targeting ARCUS Gene Editing Nuclease by AAV Eliminates Mutant Mitochondrial DNA, Demonstrating Therapeutically Meaningful Heteroplasmy Shifts In Vivo
Shoop, Wendy, et al. (2025) American Society of Gene & Cell Therapy
Definitive Preclinical Toxicokinetic and Toxicology Data Enables Advancement to Clinical Trials for a Potentially Curative Gene Editing Treatment for Chronic Hepatitis B
Harrison, Emily, et. al. (2025) Global Hepatitis Summit.
ARCUS-Mediated Excision of Exons 45-55 Leads to Functional Dystrophin and Restoration of Skeletal Muscle-Function for the Treatment of DMD
Gorsuch, C. L., et al. (2025) MDA Clinical and Scientific Conference.
AAV-meganuclease-mediated gene targeting achieves efficient and sustained transduction in newborn and infant macaque liver
Wang, L. (2022) International Conference on Ureagenesis Defects and Allied Conditions.
Targeting Hepatitis B cccDNA with a Sequence-Specific ARCUS Nuclease to Eliminate Hepatitis B Virus In Vivo
Gorsuch, C. L., (2022). European Society of Gene & Cell Therapy (ESGCT) Annual Congress.
AAV-Meganuclease-mediated Gene Targeting Achieves Efficient and Sustained Transduction in Newborn and Infant Macaque Liver
Wang L., (2022) American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.
Targeting the Hepatitis B cccDNA with a Sequence-specific ARCUS Nuclease to Eliminate Hepatitis B Virus
Gorsuch C. L., (2022) American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.