From 2003 to 2006, Cure SMA provided the very first research funding needed to begin investigation into this therapeutic approach. We would like to thank and acknowledge Cold Spring Harbor Laboratory (CSHL) and the University of Massachusetts Medical School for generating critical intellectual property for the program that was licensed to Ionis Pharmaceuticals.
We specifically thank Drs. Adrian Krainer, Yimin Hua, and colleagues at CSHL for years of dedication to and hard work on the pre-clinical development of Spinraza for SMA, as well as Drs. Ravindra Singh and Elliot Androphy for their work, funded by Cure SMA, in originally identifying the ISSN1 gene sequence, which is the sequence targeted in Spinraza.
After the pre-clinical work and early clinical testing was complete, Ionis Pharmaceuticals partnered with Biogen to launch a number of clinical trials testing Spinraza.