Cell and Gene Therapy

Background

Cell and Gene Therapy is a therapeutic modality whereby a gene is introduced into a patient’s body to generate a specific therapeutic effect. Genes are transferred into the body by the administration of vectors that are either cells, viruses, lipid nanoparticles, or other physiochemical entities.

Why Synthetic Promoters

Gene therapy vectors have a limited capacity for the therapeutic expression cassette and so derivatives of viral promoters have been used to drive strong expression of therapeutic genes. However, viral-based promoters drive uncontrollable expression both in target and non-target tissues, often introducing toxicity and immunogenicity problems for the patient. It is not possible to use endogenous tissue selective promoters to solve this issue as natural promoter sequences are too long to be inserted into gene therapy vectors and often drive sub-therapeutic expression levels of the target gene.

Our Solution

ChromatinBio design synthetic promoters using short enhancer elements to achieve cell-selective expression at levels required to mediate the therapeutic effect. We can also design promoters that respond to certain stimuli.