The pair will collaborate on Summit’s potentially breakthrough treatment for Duchenne muscular dystrophy (DMD), a muscle wasting disease that affects boys.
Sarepta is paying the UK drug developer an immediate US$40mln and US$22mln when the last patient in the company’s phase II trial is dosed.
DMD is caused by different mutations in the dystrophin gene that result in patients being unable to produce the protein that is essential for maintaining healthy muscle function.
Summit’s discovery is designed to increase the levels of another, similar, protein call utrophin.
This, it is hoped, will compensate for the lack dystrophin. The system is called utrophin modulation.
Sarepta’s chief executive, Edward Kaye, said: “Summit’s utrophin modulation technology represents a potentially promising approach to treat DMD, which may complement our current approach of exon skipping therapy.”
Duchenne muscular dystrophy, or DMD, is one of the most common, fatal genetic disorders diagnosed in children around the world.
It predominantly affects boys and it results in the progressive wasting of muscles throughout the body.
The disease has an estimated incidence of 1 in 5,000 and a patient population in the developed world of around 50,000. Patients typically don’t live beyond their late twenties.
Story by ProactiveInvestors