Gene Editing
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.
ARCUS-Mediated Excision of Exons 45-55 of the Human Dystrophin Gene using PBGENE-DMD Leads to Durable Muscle Function Improvements In Vivo as a Result of Functional Dystrophin Protein Restoration for the Treatment of Duchenne Muscular Dystrophy
Adam Mischler, et. al. (2025) American Society of Gene & Cell Therapy
Excision of the C9orf72 Hexanucleotide Repeat Expansion Using a Dual-ARCUS Gene Editing Approach Delivered by a Single AAV Reduces Neurotoxic RNA Foci and Dipeptides in an In Vivo Model of ALS
McKinstry, Spencer, et. al. (2025) American Society of Gene & Cell Therapy