Swiss drug-maker Roche said Tuesday it will license a South Plainfield biotech's spinal muscular atrophy research program, in a deal valued at up to $490 million.

Roche will license the program from PTC Therapeutics Inc., which has been working with the Spinal Muscular Atrophy Foundation since 2006 to develop a treatment for the rare genetic disorder. The foundation will continue to be an active participant in the partnership, the companies said.

Under the deal, Roche will pay $30 million up-front to license PTC's program, which currently includes three compounds in preclinical development, as well as potential back-up compounds. In addition, PTC could receive up to $460 million in milestone payments and up to double-digit royalties on any commercial sales, the companies said.

The new deal builds on existing relationships. Since 2006, the New York-based SMA Foundation has pledged $13 million in grants to support PTC's research.

Roche and PTC have collaborated since 2009 on oral therapies using PTC's Gene Expression Modulation by Small-molecules (GEMS) technology. That collaboration is initially focused on four jointly-selected central nervous system disease targets, said Cláudia Hirawat, PTC's senior vice president, corporate development.

"Having been a partner with Roche for several years, we have every confidence that the combination of our own expertise and Roche's considerable capabilities in clinical development, biomarkers and diagnostics will help us maximize the potential for this programme," said PTC President and CEO Stuart W. Peltz, in a press release announcing the collaboration.

There currently is no effective treatment for spinal muscular atrophy, which typically manifests early in life and results in muscle weakness or atrophy, according to the foundation's website. The foundation says the disorder is the leading genetic cause of death in infants and toddlers.

The disorder affects about 9,000 people in the U.S., and a similar number in Europe, according to PTC. Because of its small patient population, Roche and PTC believe any treatments they develop could qualify for "orphan" status, which in turn could speed up the time it takes to bring a product to market. Both the U.S. Food and Drug Administration and the European Medicines Agency have orphan product incentive programs designed to encourage the development of treatments for rare diseases.

The disease is caused by a defective or missing gene that results in lower levels of the survivor motor neuron protein. PTC's compounds aim to treat the underlying cause of the disorder and thus increase production of the protein.

"We found the science behind this program very compelling, with the potential to treat a currently incurable condition," said Luca Santarelli, global head of Roche Neuroscience, in a press release. "This is the essence of Roche's entire strategy, focused on solid science and high unmet clinical need, and these compounds bolster our rich pipeline in central nervous system diseases."

Representatives from Roche, PTC, and the foundation will form a joint steering committee to oversee the further development of the program. Hirawat said the research program would not exist today without the foundation's support. As the program enters a new phase, she said the foundation will continue to play a key role.

"The foundation will continue to provide the scientific support, access to research tools and reach out to the SMA community," she said. "Both companies are enthusiastic about the role the foundation will continue to play as the program advances."