(AGENPARL) – GLOBAL, mar 19 gennaio 2021
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Recent years have seen a steady acceleration in the number of cell and gene therapies (CGTs) on the market (Exhibit 1). More than 75 had been launched (including launches in individual markets, such as Japan, Russia, and South Korea) by the end of 2019. Many more are in development, offering the hope of revolutionary healthcare.
Foremost is the hope CGTs offer to patients suffering from serious, often rare, and previously unaddressed diseases. Of the 59 Phase III pipeline assets analyzed, 32 target at least one rare indication. Important too is the fact that some therapies will likely be curative, replacing lifelong treatment of chronic disease with the administration of a limited number of doses or even just one—a huge benefit to patients as well as overburdened healthcare systems. Novartis’s often-cited gene therapy Zolgensma, for example, has the potential to act as a single-dose cure for patients with spinal muscular atrophy.