After decades of slow and steady progress, cell and gene therapies are continuing to experience important breakthroughs. Gene therapies, like Luxturna
, have proven that it’s possible to not just treat symptoms, but to correct and potentially cure the underlying genetic cause of disease. Similarly, cell therapies such as cord blood and TEMCELL® HS Inj
. (the first allogeneic cell therapy launched in Japan) are proving the power that human cells have to treat disease in truly transformative ways.
Together, these cutting-edge therapies have the potential to slow disease progression, improve outcomes, and in some cases, potentially cure an array of illnesses ranging from blindness and blood cancers to inherited diseases.
However, specifically because these therapies are so new and so cutting-edge, it can be challenging for their developers to navigate through the complex clinical, commercial and regulatory hurdles that may be quite different than the challenges faced by traditional therapies.
Here, we explore some of the key clinical and regulatory questions that pharma companies should consider when developing cell and gene therapies.