Genome Plasmid

We are planning a clinical trial to treat a rare genetic disease that causes progressive dementia in children. Our therapy is based on the following: Autologous haematopoietic stem progenitor cells (HSPCs) are modified ex vivo with a lentiviral vector (LV) to express supraphysiologic levels of a secreted enzyme in myeloid progeny, in essence using gene-modified monocytes/macrophages as a durable and effective vehicle for central nervous system (CNS) enzyme delivery. In order to progress this therapy, we require the following; a plasmid to our specification, produced to FDA, IND ready standard for use in the manufacture of a lentiviral vector for clinical trial. This Plasmid will be delivered directly from the supplier to the University of Edinburgh’s partner in the project, University of California Los Angeles. (SC Ref:828196)