A phase 1/2 study of LY3884961 (PR001) an AAV9-based gene therapy for Gaucher disease type 2 – A clinical update from the PROVIDE trial.

Sarah Neuhaus, Paul Tamburri, Chester B. Whitley, Simon A. Jones, Aimee Donald, Paul Harmatz, David R. Blair, Irene Chang, Renata C. Gallagher, Deepa S. Rajan, Ozlem Goker-Alpan, Yael Beckerman, Victor A. Lopez, Daniel Hatch, Lee Shaughnessy