PERTH, Australia – On the heels of a $1.15 billion deal between Roche Holding AG and Sarepta Therapeutics Inc. for Sarepta’s gene therapy to treat Duchenne muscular dystrophy (DMD), another therapy to treat DMD is emerging from Down Under.

In a move that Sarepta Therapeutics Inc.’s president and CEO, Doug Ingram, called “transformational” for the company and “the largest licensing transaction in cell or gene therapy history,” Sarepta granted Roche Holding AG exclusive commercial rights outside the U.S. for SRP-9001, its gene therapy for Duchenne muscular dystrophy (DMD).

Perkinelmer Inc., of Waltham, Mass., has won a thumbs up from the U.S. FDA for its GSP Neonatal Creatine Kinase-MM kit. The assay is the first test approved in the U.S. for help in screening newborns for Duchenne muscular dystrophy (DMD), a rare but devastating genetic disorder. While there is no known cure for DMD, earlier screening and diagnosis could lead to improvements in quality of life by enabling earlier and more personalized treatment of symptoms. The kit’s approval opens the door for states to include DMD among the battery of tests newborns typically undergo, such as phenylketonuria, cystic fibrosis and congenital heart disease.

Solid Biosciences Inc.'s chief technology officer, Joel Schneider, said the company is "not going to leave any stone unturned" as investigators begin to explore the severe adverse event (SAE) that led to the FDA's clinical hold on the phase I/II study with SGT-001 in Duchenne muscular dystrophy (DMD).