Lysosomal Disorders

GeneTherapyLive’s Weekly Rewind – November 19, 2021

Review top news and interview highlights from the week ending November 19, 2021.

Gene-Edited, Autologous HSP Cells Correct Metabolic Activity in Hurler Syndrome

The gene-edited approach hopes to overcome with the shortcomings of autologous hematopoietic stem-cell transplantation.

Danon Disease Gene Therapy Yields Sustained Clinical Benefit

Following updated safety data, Rocket Pharmaceuticals has decided to discontinue the high-dose cohort.