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Grant: Idea to Innovation

University of British Columbia

$125,000.00

A microfluidic apparatus for the formulation of liposomal nanoparticles containing SIRNA

FY 2009-10

Summary
Genetic-based therapeutics use materials such as short pieces of DNA to either stop the expression of specific disease creating genes or to precisely replace genes missing in genetic disorders. This class of drugs has revolutionary therapeutic potential in many indications including most cancers, cardiovascular diseases, autoimmune diseases, infectious diseases, and genetic disorders. However, the development of such therapeutics is challenged by the delivery of genetic materials to diseased cells; genetic materials delivered without a vehicle are both unable to enter cells to affect biological processes and are rapidly degraded in the human body. Recent research has demonstrated that the encapsulation of genetic materials in appropriately designed lipid nanoparticles (LNP) can solve these delivery problems. However, current methods for formulating LNP containing genetic therapeutics are laborious, have limited reproducibility, are difficult to scale and cannot be readily used for many lipid compositions. We have developed a novel platform technology that enables rapid formulation of LNP siRNA systems to solve many of these issues. The apparatus currently exists as a prototype system that needs to be made user friendly and optimized for routine bench scale and intermediate scale LNP production. This proposal seeks funds to develop a prototype formulator instrument that can be used broadly by the drug development community. Our approach to the formulation of LNP encapsulated genetic therapeutics has the promise to revolutionize the genetic therapeutics market by providing a viable, much needed delivery solution. This platform technology will support a growing cluster of Canadian companies focused on genetic-based therapeutics, accelerating the time-to-market for critical therapeutics, and raising the international profile of technology in Canada. Additionally, this technology will be the basis for a Canadian based spin-off company, which will be poised to seize on the tremendous growth in genetic-based therapeutics.
Program
Idea to Innovation
Location
British Columbia, Canada
Recipient Type
Academia
Type
Grant
Source
NSERC's Awards Data

As published

Application id
387234-2009
Name nom
Cullis, Peter
Department département
Biochemistry and Molecular Biology
Organization id
2
Institution établissement
University of British Columbia
Province en
British Columbia
Province fr
Colombie-Britannique
Country en
CANADA
Country fr
CANADA
Fiscal year exercice financier
2009
Competition year année de concours
2009
Award amount
125000
Program id
I2IPJ
Program nane en
Idea to Innovation
Program name fr
De l'idée à l'innovation
Group en
Research partnerships programs
Group fr
Programmes de partenariat
Committee code
96
Committee name en
RPP Internal Decision Cttee
Committee name fr
Cté de décision interne - PPR
Area of application code
1211
Area of application group en
Advancement of knowledge
Area of application group fr
Avancement des connaissances
Area of application en
Medical and health sciences
Area of application fr
Sciences médicales et de la santé
Research subject code
1900
Research subject group en
Biomedical engineering
Research subject group fr
Génie biomédical
Research subject en
Biomedical engineering
Research subject fr
Génie biomédical
Partie
1 & 2
Nb partie
2
Application title
A microfluidic apparatus for the formulation of liposomal nanoparticles containing SIRNA
Keyword
NA
Application summary
Genetic-based therapeutics use materials such as short pieces of DNA to either stop the expression of specific disease creating genes or to precisely replace genes missing in genetic disorders. This class of drugs has revolutionary therapeutic potential in many indications including most cancers, cardiovascular diseases, autoimmune diseases, infectious diseases, and genetic disorders. However, the development of such therapeutics is challenged by the delivery of genetic materials to diseased cells; genetic materials delivered without a vehicle are both unable to enter cells to affect biological processes and are rapidly degraded in the human body. Recent research has demonstrated that the encapsulation of genetic materials in appropriately designed lipid nanoparticles (LNP) can solve these delivery problems. However, current methods for formulating LNP containing genetic therapeutics are laborious, have limited reproducibility, are difficult to scale and cannot be readily used for many lipid compositions. We have developed a novel platform technology that enables rapid formulation of LNP siRNA systems to solve many of these issues. The apparatus currently exists as a prototype system that needs to be made user friendly and optimized for routine bench scale and intermediate scale LNP production. This proposal seeks funds to develop a prototype formulator instrument that can be used broadly by the drug development community. Our approach to the formulation of LNP encapsulated genetic therapeutics has the promise to revolutionize the genetic therapeutics market by providing a viable, much needed delivery solution. This platform technology will support a growing cluster of Canadian companies focused on genetic-based therapeutics, accelerating the time-to-market for critical therapeutics, and raising the international profile of technology in Canada. Additionally, this technology will be the basis for a Canadian based spin-off company, which will be poised to seize on the tremendous growth in genetic-based therapeutics.