Periodic Reporting for period 1 - INTRASTEM (INTRASTEM: High Efficiency Delivery of Proteins, Nucleic Acids and Particles to Human Cells)
Période du rapport: 2015-07-01 au 2016-12-31
The intraSTEM proof-of-concept project aimed to position a new technology for delivering therapies and promote its commercialisation. We have invented and patented a generic system for the intracellular delivery of therapeutic molecules, termed GET (Glycosaminoglycan-binding enhanced transduction). Our system (which can be two-orders of magnitude higher efficiency than other methods) can be used with clinically relevant stem cell types, and this use is being developed for future regenerative medicine applications. The innovation potential is that GET could be a universal platform, being used in research tools, drug delivery and in cell therapy manufacture/regenerative medicine to treat disease.
Here, through strong exemplification of the key market opportunities of GET, and assessing manufacturing routes for acceptable cost of goods, we have demonstrated the potential of the opportunity. Within the testing phase (producing a technical report) we demonstrated that GET is a viable and potentially commercially attractive system to reprogram patient cells to stem cells (induced pluripotent stem cells; iPSCs) which is cutting edge technology in regenerative medicine. Furthermore the same system can be used in intact animals (in vivo) as well as with cells in culture (in vitro). This work has generated further patent fillings with new and distinct applications of the technology.
Using the grant resources we have validated a business strategy based on competitive market analyses, and freedom to operate reports. We have attended partnering meetings and through company visits we have made important stake holder contacts. Strengthening the IPR positioning and formulating the optimal commercialisation strategy has led to new industry and sector contacts.
Through engagement with interested stakeholders we have licenced the IP to a private SME. Our interaction with this partner is now guiding the exploitation strategy for other aspects of the technology as well as providing direction to exploitation of the current exemplars.
Societal and economic benefits will be enhanced from the acceleration provided by licencing and will promote the rapid clinical translation of our system. The demonstration of efficient therapeutic delivery in animals and stem cell reprogramming can be easily achieved demonstrates that GET could represent an alternative to present technologies with cost and safety concerns. We believe that after clinical translation the intraSTEM funding will yield significant socioeconomic impact in terms of saved lives and enhancing the quality of life for patients.
Here, through strong exemplification of the key market opportunities of GET, and assessing manufacturing routes for acceptable cost of goods, we have demonstrated the potential of the opportunity. Within the testing phase (producing a technical report) we demonstrated that GET is a viable and potentially commercially attractive system to reprogram patient cells to stem cells (induced pluripotent stem cells; iPSCs) which is cutting edge technology in regenerative medicine. Furthermore the same system can be used in intact animals (in vivo) as well as with cells in culture (in vitro). This work has generated further patent fillings with new and distinct applications of the technology.
Using the grant resources we have validated a business strategy based on competitive market analyses, and freedom to operate reports. We have attended partnering meetings and through company visits we have made important stake holder contacts. Strengthening the IPR positioning and formulating the optimal commercialisation strategy has led to new industry and sector contacts.
Through engagement with interested stakeholders we have licenced the IP to a private SME. Our interaction with this partner is now guiding the exploitation strategy for other aspects of the technology as well as providing direction to exploitation of the current exemplars.
Societal and economic benefits will be enhanced from the acceleration provided by licencing and will promote the rapid clinical translation of our system. The demonstration of efficient therapeutic delivery in animals and stem cell reprogramming can be easily achieved demonstrates that GET could represent an alternative to present technologies with cost and safety concerns. We believe that after clinical translation the intraSTEM funding will yield significant socioeconomic impact in terms of saved lives and enhancing the quality of life for patients.