The Jain Foundation announced the signing of a Master Service Agreement with Cellular Dynamics International (CDI). CDI will create five induced pluripotent stem cell (iPSC) lines from patients with these dystrophies. As there are currently no effective treatments for these genetic disorders, establishing model iPSC lines is an important step toward developing new drugs and therapies for these patients. Terms of the agreement were not disclosed.
Dysferlin is a protein thought to be involved in skeletal muscle repair. Genetic defects in the gene that codes for the protein result in two types of muscular dystrophy: Limb-girdle muscular dystrophy type 2B (LGMD2B) and Miyoshi muscular dystrophy 1 (MMD1). Symptoms include weakness and progressive muscle wasting, and onset typically occurs in young adults.
CDI will employ their MyCell® Products line to create LGMD2B/Miyoshi patient-derived iPSC lines. iPSC technology is based on reprogramming adult cells to a pluripotent stem cell state, whereby the resulting iPSCs, and the somatic cells derived from them, carry the genotypic backgrounds of the original donors and model the disease. A primary goal of the Jain Foundation is to use these disease models for drug discovery.
“iPSC technology promises to provide a path to better understand these devastating genetic diseases so that researchers can develop therapies,” said Plavi Mittal, President and CEO of the Jain Foundation. “CDI’s ability to make iPSCs at high quality and purity from any individual, including LGMD2B patients, will provide scientists new tools to understand and address these debilitating forms of muscular dystrophy. We look forward to working with CDI to develop the iPSC lines.”
Bob Palay, Chairman and Chief Executive Officer of CDI, said, “This is the second agreement we have entered into with an organization focused on developing new therapies for muscular dystrophy, demonstrating that research partners are confident in leveraging CDI’s technical expertise to accelerate their discoveries. CDI continues to be at the forefront of innovation and is developing additional cell types, including skeletal myoblasts that may be relevant to work supported by the Jain Foundation. We are excited that the Jain Foundation shares our vision that iPSC technology is a promising path forward to advance healthcare discoveries.”