Since 2011, the Kurt+Peter Foundation has been funding research conducted by Dr. Elizabeth McNally at Northwestern University (and previously the University of Chicago)which is pursuing exon skipping for LGMD2C. Exon skipping means jumping across “exons,” the active sections of genes.
Dr. McNally’s research has shown that a protein produced by exon skipping can cure flies with LGMD2C, treat mice with the disease, and produce the missing protein in human cells.
Currently, the Kurt+Peter Foundation is the clinical sponsor or “the drug company” developing exon skipping for LGMD2C. Prior to the Kurt+Peter Foundation funding Dr. McNally’s research no one was attempting to apply it to LGMD2C. As of 2017, the Kurt+Peter Foundation had raised and spent more than $800,000 in support of exon skipping for LGMD2C. Also in 2017, the U.S. Food and Drug Administration granted orphan drug status to the Kurt+Peter Foundation for this exon skipping program.
The Kurt+Peter Foundation is funding the development of gene therapy for LGMD2C being conducted by Nationwide Children’s Hospital.
Dr. Louise Rodino-Klapac and other Nationwide Children’s Hospital researchers have built a robust pipeline of gene therapy candidates for several LGMD types, and in January 2017, the Kurt+Peter Foundation made its first grant to support application to LGMD2C. The clinical sponsor for gene therapy for LGMD2C will be Myonexus Therapeutics.