Elk Grove Village, IL. May 10, 2012.

This new program will focus on optimizing small molecule drug candidates which increase the SMA back-up gene in order to make them ready for human clinical trials. It is well demonstrated that increasing production from the back-up gene can lead to improvement in mouse models of SMA. Small molecule drug candidates have particular advantages: mainly ease of use by oral dosing and the ability to clear the drug quickly from the body. Dr. Peter Schultz, a renowned chemist and successful biotech leader, will be leading this new research effort.

In addition to founding the California Institute for Biomedical Research where the Families of SMA funded project will take place. Dr. Schultz also was Institute Director at the Genomics Institute of the Novartis Research Foundation (GNF) from 1999 to 2010. Dr. Schultz has founded multiple biotech companies, including Affymax Research Institute, Syrrx, Kalypsys, Phenomix, Symyx Technologies, Ilypsa, Ambrx and...