With the advent of disease-modifying SMA therapies such as Evrysdi (risdiplam) and Spinraza (nusinersen), regenerative medicine — which aims to restore damage caused by the disorder — has become a key research and development target.
“In neuromuscular disease, correcting the underlying cause is just the first step,” Loren Eng, president of the nonprofit SMA Foundation, said in a press release. “Patients need therapies to help restore lost function. We are excited to expand our partnership with PTC Therapeutics to empower researchers to address this therapeutic gap in neuromuscular disorders with an emphasis on SMA.”
PTC and the SMA Foundation, along with Roche, have collaborated in the past, most notably on the development of Evrysdi. The oral liquid therapy boosts the ability of the SMN2 gene to produce functional copies of the SMN protein, which is lacking in SMA, promoting the healthy growth and survival of motor neurons — those gradually lost in patients as the disease progresses.
In clinical trials, participants using Evrysdi saw improvements in swallowing, breathing, survival, and motor function. This contributed to the medication’s approval in the United States, Brazil, Russia, Chile, Ukraine, South Korea, and Georgia, with at least 30 other countries, including those in Europe, reviewing the therapy for possible approval.
“Our longstanding partnership with PTC lets us harness our research capabilities and PTC’s drug discovery and development expertise and access to core technology platforms, all of which supported the recent approval of Evrysdi (risdiplam) in SMA,” Eng added. “We look forward to our initial funding of new projects under the collaboration with PTC.”
The collaborators committed to an initial investment of up to $60 million for academic institutions and other collaborators, to be managed by the SMA Foundation, towards the discovery and development of promising new regenerative treatments.
“We have had an extremely productive partnership with the SMA Foundation for well over a decade and are excited to continue this collaboration to investigate regenerative medicine to find new therapies for patients with SMA and other neuromuscular disorders,” said Stuart W. Peltz, PhD, CEO of PTC Therapeutics. “The SMA Foundation has been the leader in supporting SMA research and we look forward to doing the critical work to accelerate the next generation of therapeutics for these patients.”
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