CureDuchenne has launched a new drug discovery and development initiative to identify and develop a pipeline of therapies to treat Duchenne muscular dystrophy.
The initiative, dubbed CureDuchenne Ventures, is based on the company's venture philanthropy model, in which the organization provides early stage funding to biotechnology, pharmaceutical firms and academic investigators to help develop breakthrough therapies to treat Duchenne.
CureDuchenne Ventures will focus on venture philanthropists, foundations, and donors with an interest in scientific research for rare diseases like Duchenne.
The first research project to be funded through CureDuchenne Ventures is a collaboration with biopharmaceutical firm, Lexicon Pharmaceuticals.
CureDuchenne said Lexicon's experimental drug, LX2931, which is in clinical development as a potential treatment of rheumatoid arthritis and autoimmune disease, is currently being evaluated for its potential to initiate muscle regeneration in studies with the mdx mouse model of Duchenne.
US-based CureDuchenne intends to raise $5m to support clinical development of LX2931 as a potential treatment for Duchenne, with clinical studies scheduled as early as 2014.
CureDuchenne chief scientific officer Michael Kelly said 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.
"CureDuchenne Ventures will power our ability moving forward to support the most promising therapies and ensure that multiple avenues of research are explored," Kelly added.
"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."
