Periodic Reporting for period 1 - TraffikGene (Peptide Dynamic Amphiphiles for Gene Therapy and Macromolecular Delivery)
Berichtszeitraum: 2019-07-01 bis 2021-06-30
ERC-PoC-TraffikGene has been focused on exploring the technology transference potential of the results obtained during ERC-Stg-DYNAP in the fields of gene therapy, cytosolic delivery, antibody transport, immunotherapy and cell targeting. In this regard we have obtained refined peptide vehicle libraries that we have thoroughly evaluated for in vitro and in vivo applications. In a high-throughput screening assay carried out in collaboration, we have clearly identified tens of amphiphilic peptidic candidates that outperformed current benchmark commercial reagents for in vitro delivery of nucleic acids (e.g. siRNA, pDNA, mRNA) and the ribonucleoprotein Cas9 (CRISPR/Cas). The best performing candidates were evaluated externally (beta testing) in real market conditions to carry out pDNA delivery for the expression of big amounts of proteins. Additionally, we have evaluated the in vivo delivery of mRNA in mice using the best amphiphilic vehicles. Studies with mRNA encoding for model proteins, such as luciferase, have demonstrated excellent in vivo delivery efficiency following the administration of our peptide-mRNA formulations via different routes of injection (i.m. s.c. i.d. and i.v.) with no signs of toxicity. Remarkably, for systemic i.v. administration, we have discovered a spleen targeted biodistribution, a very interesting result towards the application of this novel delivery technology in the immunotherapy area.
Motivated by these promising results, we have started technology transfer activities to evaluate the potential fitness for reaching the market. In this sense, we have carried out a market study that covered the technological state of the art in the field of gene delivery, the main stakeholders (competitors and potential industrial partners) and our target product profile. This market study has been complemented with an external validation of our delivery vehicles, which we have done in collaboration with potential end users (academic researchers) and companies.
The scientific breakthroughs, which will soon be covered by a patent application and then published on a scientific journal, and the preliminary activities for market transference, clearly indicate that the developed technology holds a great promise in the growing field of gene therapy and nucleic acid therapeutics, which will be our next target for commercialisation.
Motivated by these promising results, we have started technology transfer activities to evaluate the potential fitness for reaching the market. In this sense, we have carried out a market study that covered the technological state of the art in the field of gene delivery, the main stakeholders (competitors and potential industrial partners) and our target product profile. This market study has been complemented with an external validation of our delivery vehicles, which we have done in collaboration with potential end users (academic researchers) and companies.
The scientific breakthroughs, which will soon be covered by a patent application and then published on a scientific journal, and the preliminary activities for market transference, clearly indicate that the developed technology holds a great promise in the growing field of gene therapy and nucleic acid therapeutics, which will be our next target for commercialisation.