Promethera is looking to advance selected development candidates from its portfolio together with industry partners to increase the likelihood of success in development. These activities include the use of genetically modified liver-derived stem cells in hemophilia and other diseases, where Promethera’s cells can be used as a long-term, endogenous delivery system for the partner’s therapeutic candidate.
Gene-Transfer Augmented Therapeutic Cell Lines
Promethera has established a combo cell-gene therapy platform that can give rise to promising product candidates in hemophilia A and B and potentially other indications. These approaches use the clinically validated HepaStem product line.
Hemophilia is a genetic disorder that impairs the body's ability to make blood clots. Hemophilia patients bleed longer after an injury, bruise easily, and are an increased risk of internal bleeding.
Hemophilia remains a disease in search for a cure. While gene therapy has provided new hope for hemophilia patients, issues surrounding the durability of expression and immune responses against gene transfer vectors remain.
Promethera is pursuing a potential solution to this problem using HepaStem cells which are genetically engineered ex vivo with Factor VIII or Factor IX and then applied via intravenous infusion.
HepaStem consists of liver-derived mesenchymal stem cells (MSCs progenitor cells) isolated from normal adult human liver tissue. Safety has been shown in Phase I/II clinical trial in 20 young patients. Promethera has successfully generated HepaStem cells which highly express Factor IX. Following intravenous infusion in a hemophilia patient, homing of these cells to the liver as well as to a site with hemophilia-related arthropathy has been observed.
This cell-based gene therapy approach is currently in pre-clinical stage and is expected to allow physiological FVIII/FIX production as well as cell re-administration because of the low immunogenicity of MSCs.
For more information, please contact our Business Development team.