In a recent article in IPT magazine, Synpromics’ founder and Chief Scientific Officer Dr. Michael L Roberts discusses the current obstacles to overcome in optimising gene therapies. Despite the many advances made in gene delivery to target cells, and our understanding of the role of gene regulation in a cell’s molecular phenotype, use of gene therapy is still in its early stages.
Synpromics is a company addressing one of the remaining issues in effective cell and gene therapy: sufficient expression of the desired gene to the required levels and restricted to the appropriate cells. Furthermore, to allow the therapeutic effect to last a lifetime, silencing of gene expression must be avoided and the long-term immune responses to the therapeutic cassette need to be minimised. This can be achieved by careful design of promoters driving therapeutic gene expression. Synpromics has developed a means to unravel which combinations of transcription factors control cell fate, and has developed a methodology to create libraries of promoter candidates with the potential to be active only in specific cells and certain environments. Using this specificity, the result is a novel promoter that tightly controls gene expression in the particular cell type and condition of interest, and comprises a sequence constituting a novel combination of enhancer elements that does not exist in nature and can thus be patented.
With our current understanding of the cell’s molecular phenotype and the engineering biology techniques available to harness them, Synpromics’ technologies will be able to take cell and gene therapy to the next stage.
Read the full article here.