Dear Duchenne Community Newsletter:

First patient has been dosed in the FUNCTION-DMD clinical trial, a major step forward for our DMD gene editing program.

by Chris Robinson

First evidence of elimination and inactivation of cccDNA in liver biopsies collected from patients with chronic hepatitis B treated with PBGENE-HBV

by Chris Robinson

PBGENE-DMD gene editing drives safe, efficacious, and durable functional improvement in a humanized Duchenne muscular dystrophy mouse model

by Chris Robinson

Dear Duchenne Community Newsletter:

First clinical trial site is now open at Arkansas Children's Hospital (Little Rock, AR) with Dr. Aravindhan Veerapandiyan as the study’s principal investigator.

by Chris Robinson

PBGENE-DMD gene editing treatment leads to safe and long-term functional improvement in humanized DMD-disease mouse model

by Chris Robinson

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

by Chris Robinson

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