OCAT
Hoy era la presentación de Lupus del cell and gene teraphy
Alguno sabe si ha sido ya o a que hora es?
Edito: a las 14:30
Erin A Kimbrel
Pluripotent stem cells (PSCs) have important clinical potential in regenerative medicine given their ability to differentiate into any cell type in the body. While inefficient differentiation and concerns over potential tumorigenicity have hindered their clinical development, the first wave of PSC-based therapies is now being tested in clinical trials and showing safe application in humans. Additional PSC-derived products are being developed for treating a variety of diseases, including autoimmune disorders. Through a unique differentiation and manufacturing process, we have established a platform for the generation of highly potent immunomodulatory cells termed hemangio-mesenchymal cells, or HMCsTM from pluripotent stem cells. Preclinical data show strong therapeutic activity for HMCs in models of lupus nephritis, Crohn’s, multiple sclerosis, and autoimmune uveitis, principally through the secretion of paracrine-acting factors that alter the behavior of innate and adaptive immune cells. Starting from a pluripotent stem cell source, HMCs are both youthful and replenishable, representing the earliest stage of biological development. Preclinical evidence shows that these attributes afford HMCs greater potency than mesenchymal stromal cells, which are derived from primary tissue sources like bone marrow and umbilical cord. Moreover, banks of genotypically identical HMCs can be generated on a commercial scale without the need for extended in vitro culture or use of multiple donors, which facilitates a consistent final product. Various stages in the development of a pluripotent stem cell-based product like HMCs- from disease model testing to quality control in manufacturing to safety and biodistribution - will be addressed.
Key points:
1. The current status of pluripotent stem cell (PSC)-based products in clinical trials
2. Preclinical evaluation of HMCs, a PSC-based product for treating autoimmune diseases
3. A comparison between the use of PSCs and primary tissues as sources for cell-based products
4. GMP lot release considerations and safety testing for PSC-based therapies