PBGENE-HBV, a First-in-class Gene Editing Therapy for Chronic Hepatitis B, Demonstrates Safety and Antiviral Activity in Early Cohorts

by Chris Robinson

Treatment with PBGENE-DMD results in durable improvements in muscle function over time through increased dystrophin expression and dystrophin-positive cells

by Chris Robinson

High-efficiency homology-directed insertion into the genome using the engineered homing endonuclease ARCUS

by Chris Robinson

PBGENE-HBV Chronic Hepatitis B Program

by Chris Robinson

Initial safety data from ELIMINATE-B, the first clinical trial of a gene editing treatment for chronic hepatitis B

by Chris Robinson

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

by Chris Robinson

Systemic Delivery of a Mitochondria-Targeting ARCUS Gene Editing Nuclease by AAV Eliminates Mutant Mitochondrial DNA, Demonstrating Therapeutically Meaningful Heteroplasmy Shifts In Vivo

by Chris Robinson

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

by Chris Robinson

Initial safety data from ELIMINATE-B, the first clinical trial of a gene editing treatment for chronic hepatitis B.

by Chris Robinson

Definitive Preclinical Toxicokinetic and Toxicology Data Enables Advancement to Clinical Trials for a Potentially Curative Gene Editing Treatment for Chronic Hepatitis B

by Chris Robinson

ARCUS-Mediated Excision of Exons 45-55 Leads to Functional Dystrophin and Restoration of Skeletal Muscle-Function for the Treatment of DMD

by Chris Robinson

High-efficiency homology-directed insertion into the genome using ARCUS nucleases.

by Chris Robinson

Shifting Heteroplasmy with PBGENE-PMM: Gene Editing Therapy for m.3243A>G Associated Mitochondrial Myopathy

by Chris Robinson

Preclinical safety data for PBGENE-HBV gene editing program supports advancement to clinical trials as a potentially curative treatment for chronic hepatitis B

by Chris Robinson

Optimization of targeted gene insertion using ARCUS nucleases

by Chris Robinson