Jeffrey Sevigny, Olga Uspenskaya, Laura Dean Heckman, Li Chin Wong, Daniel A Hatch, Ambika Tewari, Rik Vandenberghe, David J Irwin, Dario Saracino, Isabelle Le Ber, Rebekah Ahmed, Jonathan D Rohrer, Adam L Boxer, Sebastian Boland, Patricia Sheehan, Alissa Brandes, Suzanne R Burstein, Benjamin M Shykind, Sitharthan Kamalakaran, Carter W Daniels, E David Litwack, Erin Mahoney, Jenny Velaga, Ilan McNamara, Patricia Sondergaard, Syed A Sajjad, Yvonne M Kobayashi, Asa Abeliovich, Franz Hefti
GRN mutations cause progranulin haploinsufficiency, which eventually leads to frontotemporal dementia (FTD-GRN). PR006 is an investigational gene therapy delivering the granulin gene (GRN) using an adeno-associated virus serotype 9 (AAV9) vector. In non-clinical studies, PR006 transduced neurons derived from induced pluripotent stem cells of patients with FTD-GRN, resulted in progranulin expression and improvement of lipofuscin, lysosomal and neuroinflammation pathologies in Grn-knockout mice, and was well tolerated except for minimal, asymptomatic dorsal root ganglionopathy in non-human primates...
May 14, 2024: Nature Medicine