Swiss pharmaceutical giant Roche, PTC Therapeutics and the SMA Foundation, announced today a licensing agreement for PTC's Spinal Muscular Atrophy (SMA) programme. The deal puts Roche in a race against another giant Novartis, which is also collaborating with SMA Foundation.
PTC Threapeutics have identified three different compounds that each increases SMN protein levels and extends median survival of severe SMA mice. These compounds are orally bioavailable and can penetrate the blood-brain barrier, but the mechanism by which they alter SMN splicing has not yet been described.
Under the terms of the agreement, Roche gains an exclusive worldwide license to PTC's SMA programme, which includes three compounds currently in preclinical development, as well as potential back-up compounds. Development will be overseen by a joint steering committee comprised of members from Roche, PTC and the SMA Foundation.
"This collaboration brings us one step closer to developing a treatment for a condition that has a profound effect on the lives of many thousands of children and their parents worldwide," said Dinakar Singh, Chairman of the SMA Foundation. "We are very optimistic that, by building on the pioneering efforts of PTC Therapeutics, Roche can help us realise what we have been working so hard to achieve."
Luca Santarelli, Global Head of Roche Neuroscience, said: "We found the science behind this programme very compelling, with the potential to help treat a currently incurable condition. 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. As an established partner of Roche, we already have experience with PTC's scientific approach. Together with the involvement of the SMA Foundation, we now have the opportunity to make a significant impact in the treatment of SMA."
PTC Threapeutics have identified three different compounds that each increases SMN protein levels and extends median survival of severe SMA mice. These compounds are orally bioavailable and can penetrate the blood-brain barrier, but the mechanism by which they alter SMN splicing has not yet been described.
Under the terms of the agreement, Roche gains an exclusive worldwide license to PTC's SMA programme, which includes three compounds currently in preclinical development, as well as potential back-up compounds. Development will be overseen by a joint steering committee comprised of members from Roche, PTC and the SMA Foundation.
"This collaboration brings us one step closer to developing a treatment for a condition that has a profound effect on the lives of many thousands of children and their parents worldwide," said Dinakar Singh, Chairman of the SMA Foundation. "We are very optimistic that, by building on the pioneering efforts of PTC Therapeutics, Roche can help us realise what we have been working so hard to achieve."
Luca Santarelli, Global Head of Roche Neuroscience, said: "We found the science behind this programme very compelling, with the potential to help treat a currently incurable condition. 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. As an established partner of Roche, we already have experience with PTC's scientific approach. Together with the involvement of the SMA Foundation, we now have the opportunity to make a significant impact in the treatment of SMA."
Comments
Post a Comment