National nonprofit CureDuchenne has launched CureDuchenne Ventures, a new initiative to identify and develop a robust pipeline of therapies to treat Duchenne, the most common and lethal form of muscular dystrophy. This drug discovery and development initiative is based on CureDuchenne's venture philanthropy model, in which the organization provides early stage funding to biotechnology, pharmaceutical companies and academic investigators to help develop breakthrough therapies to treat Duchenne.
Currently, there is no treatment for Duchenne. Duchenne is a progressive muscle-wasting disease that impacts approximately 1 in 3,500 boys. Boys with Duchenne are usually diagnosed before the age of five and are in a wheelchair by age 12; most don't survive their mid-20s.
CureDuchenne Ventures will focus on venture philanthropists, foundations and donors with an interest in scientific research for rare diseases, such as Duchenne. The first research project to be funded through CureDuchenne Ventures is a collaboration with biopharmaceutical company Lexicon Pharmaceuticals. Lexicon's experimental drug, LX2931, which is in clinical development as a potential treatment of rheumatoid arthritis and autoimmune disease, currently is being evaluated for its potential to initiate muscle regeneration in studies with the mdx mouse model of Duchenne. CureDuchenne plans to raise $5 million to support clinical development of LX2931 as a potential treatment for Duchenne, with clinical studies slated to begin as early as 2014.
"Multiple technologies exist to repair, restore or replace the missing protein in Duchenne; however, the limiting factor in getting treatments out of the laboratory and into the clinic is funding," said Michael G. Kelly, chief scientific officer, CureDuchenne. "CureDuchenne Ventures will power our ability moving forward to support the most promising therapies and ensure that multiple avenues of research are explored. Multiple therapeutic strategies, working together, will be needed to effectively treat the whole disease, and the purpose of our drug discovery and development initiative is to identify and support those programs that would critically impact the various components of the disease."
CureDuchenne already has raised more than $11 million toward promising research aimed at treating and curing Duchenne. CureDuchenne has funded seven research projects that have advanced to human clinical trials. CureDuchenne's research investments have leveraged more than $100 million from biotech and pharmaceutical companies, and other foundations to fund research leading to a cure.