Vectalys’s platform is based on retroviral (RV), lentiviral (LV) and adeno-associated virus (AAV) based vectors that efficiently introduce human gene sequences into a wide variety of human cells to knock-in or knock-down specific proteins.

The Human Genome sequencing has provided a revolution in biology and biotechnology. It has increased our ability to analyze, manipulate, and modify genes and to engineer organisms, providing numerous opportunities for applications. Within the human genome is an estimated total of 6000 genes that have a direct impact on the diagnosis and treatment of human genetic diseases. A central challenge in post-genomic drug discovery is selection of relevant therapeutic targets from a large pool of candidates both in vitro and in vivo, so that resources are invested productively.

Gene transfer opens the way to develop cell and animal models useful to reach this challenge by transferring target genes into functional cells and tissues. Because primary cells are non permissive towards classical gene transfer technologies, viral based vectors derived from lentiviruses or AAV are the only efficient technology to achieve high expression of genes or silencing sequences in cells or tissues.

Vectalys is a gene delivery platform providing viral recombinant vector sfor:

• gene silencing
• gene over-expression
• cell-based assay development
• protein expression and production
• Preclinical trials for gene therapy
• creation of animal models