In gene therapy, synthetic promotors – used to regulate gene activity and precisely control protein production – deliver therapeutic genes to immune cells such as microglia, macrophages, and T-cells.
Synpromics said the research collaboration provides UCL access to its synthetic promotors, which “can be used to drive more efficient expression of therapeutic genes in a more selective manner”, to bolster the Institute's cell therapy programmes.
The promotors could improve “the efficacy of their therapeutic vectors and safety profile by limiting off-target expression,” a Synpromics spokesperson told us.
According to the firm, the promotors could also help the development of CAR-T therapies, which use immune effector cells (T-cells).
“The promoters developed in this collaboration can be used to more effectively drive expression of the Chimeric Antigen Receptor in the T-cell, or to arm the CAR-T cell to express other immunomodulatory proteins that could enhance the potency of the therapy,” the spokesperson said.
Biggest challenge?
The primary hurdle in the collaboration stems from the many difference cell lineages that comprise the haematopoietic system, we were told.
“The biggest challenge is delineating the transcription profile that underpin each lineage and leveraging that information to build synthetic promoters that are highly selective for each cell lineage.
“Thus, given the paucity of cell selective promoters for use in the haematopoietic system, success of this project would yield a selection of tools that could significant advance the development of more effective cell therapies,” the spokesperson said.
Synpromics CSO Michael Roberts said the firm’s technology is particularly suitable for developing gene and cell therapies for blood-based disorders.
“We’re able to design promoters that are active in any cellular lineage of the haematopoietic system by leveraging the subtle changes in transcription profiles that are evident in the different cell populations present in the blood,” he said.
The Scottish firm moved to a larger facility last year near Edinburgh, UK, which added over 5,000 sq. ft. of laboratory and office space.