CAR T Immunotherapy
Preliminary Safety and Efficacy of PBCAR0191, an Allogeneic, Off-the-Shelf CD19-Targeting CAR-T Product, in Relapsed/Refractory (r/r) CD19+ NHL
Shah, B.D. et al. American Society of Clinical Oncology 2021 Annual Meeting.
Initial Findings of the Phase 1 Trial of PBCAR0191, a CD19 Targeted Allogeneic CAR T Cell Therapy
Jacobson C.A. et al. American Society of Hematology 2019 Annual Meeting.
Off the Shelf T Cell Therapies for Hematologic Malignancies
McCreedy, B. J. et al. Best Pract Res Clin Haematol. 2, 166-175 (2018).
Integration of a CD19 CAR Into the TCR Alpha Chain Locus Streamlines Production of Allogeneic Gene-Edited CAR T Cells
Macleod, D.T. et al. Molecular Therapy. 4, 949-961 (2017).
In Vivo Gene Editing
Mitochondrial Targeted Meganuclease as a Platform to Eliminate Mutant mtDNA In Vivo
Zekonyte, U. et al. Nat Commun. 12, 3210 (2021).
Rho 1-2 Meganuclease, an Allele-Specific Gene Editing Therapy, Rejuvenates Rod Photoreceptor Structure and Function in a Pig Model of adRP
Jalligampala, A. et al. Association for Research in Vision & Ophthalmology 2021 Meeting.
Translation of an AAV-Delivered Gene Editing Approach for Transthyretin Amyloidosis in Animal Models
Greig, J.A. et al. American Society of Gene and Cell Therapy 2021 Annual Meeting.
Long-Term Stable Reduction of Low-Density Lipoprotein in Nonhuman Primates Following In Vivo Genome Editing of PCSK9
Wang, L. et al. Molecular Therapy. 6, 2019-2029 (2021).
A Gene Editing Approach to Eliminate Hepatitis B Virus Using ARCUS Meganucleases
Gorsuch, C. et al. American Society of Gene and Cell Therapy 2020 Annual Meeting.
Engineering a Self-inactivating Adeno-associated Virus (AAV) Vector for ARCUS Nuclease Delivery
Li, H. et al. American Society of Gene and Cell Therapy 2020 Annual Meeting.
Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol
Wang, L. et al. Nat Biotechnol. 8, 717-725 (2018).