Currently, the cell and gene therapy (CGT) field is on track for a serious capacity shortage. In particular, there is a significant shortage of manufacturing capacity at the commercial scale. Although nearly 90% of cell and gene therapy developers would prefer to use contract manufacturing organizations (CMOs), relevant CMO capacity is not yet available. [Read more…]
Mesenchymal stem cells (MSCs) are a type of non-controversial, adult stem cell that prefers to differentiate into the specialized cells found in skeletal tissues, such as cartilage cells (chondrocytes), bone cells (osteoblasts), and fat cells (adipocytes). In contrast, stem cells derived from embryos are capable of developing into all the different types of cells found in our body, which makes them more versatile than MSCs, but also more controversial. Induced pluripotent stem cells also exhibit greater pluripotency, but as modified cells, they come with a greater set of regulatory challenges. [Read more…]
Despite progress involving the use of induced pluripotent stem cells (iPSCs) within disease modeling and drug discovery applications, it will be a long path to achieve the broad-scale use of iPSC-derived cell types in human patients. [Read more…]
To date, 53 RMAT (Regenerative Medicine Advanced Therapy) designations have been publicly announced. However, the FDA states it has received 165 requests and issued 62, which means that a handful are not yet public knowledge.
Sponsors of cell and gene therapies are eligible to obtain an RMAT designation from the U.S. FDA if their product is intended to treat serious or life-threatening diseases and there is preliminary clinical evidence that it can address unmet medical needs.
Thus far, only three companies (Abeona Therapeutics, Athersys, and Rocket Pharmaceuticals) have received two RMAT designations from the U.S. FDA.
What is the difference between an allogeneic and autologous treatment? Whether it is a stem cell treatment, a bone marrow transplant, or another type of cellular therapy, allogeneic means “from another person” while autologous means “from oneself.”
Let’s dig into this in more detail and explain why both of these approaches are commonly used within medicine.