Synpromics has entered into another gene therapy collaboration with UCL. This time around the aim is to apply Synpromics’ synthetic promoter expertise to the development of gene therapies against diseases affecting the blood.
Working with Adrian Thrasher, M.D, Ph.D., and his clinical research group at the UCL Great Ormond Street Institute of Child Health, Synpromics plans to create tissue-specific promoters. These promoters will be tailored to use in lymphoid, myeloid, microglia and other specialized immune cells. Synpromics sees the promoters being used in gene-modified cell therapies, such as treatments that use microglia cells to deliver proteins.
Synpromics CSO Michael Roberts, Ph.D., thinks this application is right in the company’s wheelhouse.
“Our technology is particularly suitable for developing gene and cell therapies for blood-based disorders,” Roberts said in a statement. “We’re able to design promoters that are active in any cellular lineage of the hematopoietic system by leveraging the subtle changes in transcription profiles that are evident in the different cell populations present in the blood.”
News of the collaboration comes two months after Synpromics teamed up with another group at UCL to work on a gene therapy for young-onset Parkinson's disease.
The two projects are going after very different diseases, but they share underlying characteristics that are indicative of Synpromics’ strengths and its strategy for leveraging them. In both cases, attempts to develop treatments for the diseases could be undermined by loose regulation of gene expression. Synpromics thinks its promoters bypass this pitfall and thereby open new treatment possibilities.
Roberts and his colleagues are putting this idea to the test by striking deals to seed the development of gene therapies that require tight regulation of gene expression to work. UCL, an active player in gene therapy development in its own right, is a beneficiary of this business development strategy.