GlaxoSmithKline (GSK) has announced the formation of a new specialised unit to research and develop treatments for rare diseases.
The alliance with Prosensa will focus on nucleic acid-based therapeutics, correcting gene expression in diseases with large unmet medical needs. This includes developing four RNA-based compounds intended to treat different subpopulations of patients with Duchenne muscular dystrophy.
As part of the agreement with Japan’s JCR Pharmaceuticals, GSK has obtained global rights to a number of enzyme replacement therapies with the potential to treat orphan diseases such as Hunter syndrome, Fabry disease and Gaucher disease.
GSK president of Asia Pacific and chairman of Japan Marc Dunoyer said that in addition to the company’s existing discovery effort, alternative opportunities need to be explored to make treatments available for rare diseases.
“Over time, this new unit has the potential to deliver multiple therapies responding to high medical needs of underserved populations of patients,” Dunoyer said.
According to GSK, over 5,500 rare diseases have been identified, of which less than 10% are being treated.