Gene editing strategies
The ability to deliver DNA endonucleases –ZFNs, TAL, CRISPR- which specifically bind to a DNA sequence of choice and induce a break into the genome, brings in situ gene correction. All of these strategies entail a mechanism that requires only transient expression of the nuclease complex.
Gene editing strategies are being developed mainly for use in ex vivo approaches and will be tested with LentiFlash for blood and liver disorders.
Immunotherapy refers to therapies that take advantage of the immune system in order to attempt to eradicate tumor cells or viruses. New approaches require that patient cells be genetically modified either to induce an immune response or to improve their targeting and cytotoxicity. To make these new gene therapies viable in the clinic, a reliable method for introducing transgenes safely and effectively is essential. RNA delivery and lentiviral vectors are respectively emerging as safer and effective options to respectively deliver nucleases machineries and chimery antigen receptor into lymphocytes.
Gene therapy strategies
Many diseases caused by an absent or abnormal gene, lead to a missing or defective protein expression and a defective cell phenotype. As a result, a lot of gene therapy approaches based on the addition of the missing gene in patient cells, to restore the normal phenotype, are underway.
Stand-alone clinical programs
Flash therapeutics will take in charge standalone indications that will use ex vivo CRISPR/Cas9 gene-editing therapeutic strategy.
For these programs, the delivery technology must guarantee:
- a transient expression to preserve the cell genome from off-target effects of nucleases, and avoid cellular toxicity and immunogenicity,
- a clinically suitable delivery of the editing machinery that leads to a controlled expression efficiency of the editing machinery in all the target cell population without damaging target cell’s phenotype or viability.
Perfectly fitting with the two conditions above, we naturally chose to use LentiFlash to develop our standalone programs. Blood and hepatic fields are under investigation with clinical partners specialized in such fields.
Partnered clinical programs
While developing our own therapeutic programs, we look forward to negotiating partnerships and licensing agreements with pharmaceutical or biotechnological companies for the utilization, further development and global commercialization of the proprietary LentiFlash delivery system.
The potential field of application of LentiFlash is wide, encompassing any disease involving a cell type that can be effectively transduced by a lentiviral vector in vivo or ex vivo and where a transient transgene expression can lead to a durable therapeutic benefit. In this context, immunotherapy, gene editing, regenerative medicine applications appear as natural primary targets, with LentiFlash offering a highly safe and easy-to-use solution.