The Jain Foundation, a nonprofit organization whose mission is to cure muscular dystrophies caused by dysferlin protein deficiency, has signed a Master Service Agreement with Cellular Dynamics International (CDI), a developer of next-generation stem cell technologies for drug development, cell therapy, tissue engineering and organ regeneration.
CDI will create five induced pluripotent stem cell (iPSC) lines from patients with these dystrophies. As there currently are no effective treatments for these genetic disorders, establishing model iPSC lines is an important step toward developing new drugs and therapies for these patients. Financial terms 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.”
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 experience to accelerate their discoveries. CDI is developing additional cell types, including skeletal myoblasts, that may be relevant to work supported by the Jain Foundation.”