Publications

Peer Reviewed Work Supporting Our Therapeutic Approach

Allogeneic CAR T

Jacobson C.A. et al. Initial Findings of the Phase 1 Trial of PBCAR0191, a CD19 Targeted Allogeneic CAR-T Cell Therapy. American Society of Hematology 2019 Annual Meeting, Orlando, FL.

McCreedy, B. J. et al. Off the shelf T cell therapies for hematologic malignancies. Best Pract Res Clin Haematol. 2, 166-175 (2018).

Macleod, D.T. et al.  Integration of a CD19 CAR into the TCR alpha chain locus streamlines production of allogeneic gene-edited CAR T cells. Mol Ther. 4, 949-961 (2017)PDF

 

In vivo gene correction

Gorsuch et al. A Gene Editing Approach to Eliminate Hepatitis B Virus Using ARCUS Meganucleases. American Society of Gene and Cell Therapy 2020 Annual Meeting.

Li et al. Engineering a Self-inactivating Adeno-associated Virus (AAV) Vector for ARCUS Nuclease Delivery. American Society of Gene and Cell Therapy 2020 Annual Meeting.

Wang, L. et al.  Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol. Nat Biotechnol. 8, 717-725 (2018).

-Press release from Penn Medicine

-Write up in Science