Poster
Treatment with PBGENE-DMD results in durable improvements in muscle function over time through increased dystrophin expression and dystrophin-positive cells
Mischler, Adam. et al. (2025) World Muscle Society
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
Mischler, Adam, 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
Initial safety data from ELIMINATE-B, the first clinical trial of a gene editing treatment for chronic hepatitis B.
Jucov, Alina, et al. (2025) The European Study of the Association of the Liver Conference.
High-efficiency homology-directed insertion into the genome using ARCUS nucleases.
Mischler, A., et al. European Society of Gene & Cell Therapy (ESGCT) 2024 Annual Congress.
Preclinical safety data for PBGENE-HBV gene editing program supports advancement to clinical trials as a potentially curative treatment for chronic hepatitis B
Harrison, E. et al. European Association for the Study of the Liver (EASL) Congress 2024.
Optimization of targeted gene insertion using ARCUS nucleases
Shoop W, et al., Precision Genome Engineering 2023 Conference
Specific elimination of m.3243A>G mutant mitochondrial DNA using mitoARCUS
Shoop W., et al., EuroMIT Conference 2023 Conference
Shifting m.3243A>G heteroplasmy with PBGENE-PMM: Gene editing therapy for primary mitochondrial myopathy
Shoop W, et al., Mitochondrial Medicine – Therapeutic Development 2024 Conference
ARCUS-mediated excision of the “hot spot” region of the human dystrophin gene results in functional improvement in a mouse model of Duchenne muscular dystrophy (DMD)
Owens, G., et al. European Society of Gene and Cell Therapy (ESGCT) 2023 Annual Congress.
Unique features of ARCUS nucleases enable high efficiency, targeted gene insertion in vivo
Gorsuch, C., et al. European Society of Gene and Cell Therapy (ESGCT) 2023 Annual Congress.
Preclinical efficacy and safety of ARCUS-POL nucleases for chronic hepatitis B: a potentially curative strategy
Harrison E., et al. American Association for the Study of Liver Diseases (AASLD) The Liver Meeting 2023.
Effective Cell Dose and Functional Attributes of Azercabtagene Zapreleucel (Azer-cel; PBCAR0191) Associated with Allogeneic CAR T-Cell Safety and Efficacy in Patients with Relapsed/Refractory B-Cell Lymphoma
Jacobson, C.A., American Society of Hematology (ASH) 2022 Annual Meeting
ARCUS gene editing of Apolipoprotein C3 results in substantial reduction in serum triglycerides in vivo
Shoop W., et al. European Society of Gene & Cell Therapy (ESGCT) 2022 Annual Congress.
Specific elimination of m.3243A>G mutant mitochondrial DNA using mitoARCUS in cultured cells and a novel xenograft mouse model
Shoop, W., European Society of Gene & Cell Therapy (ESGCT) 2022 Congress.