C12218: Efficient Delivery Vehicle for Controlled Gene Knockdown
Novelty:
This technology comprises the use of bioreducible polymer based nanoparticle system that can release siRNA cargo to the cytoplasm of cells to knockdown target gene expression in tissues.
Value Proposition:
Gene therapy is a developing medical field and there is a need for systems that can rapidly and efficiently deliver therapeutic products to the site of action. siRNA based gene therapy is one such approach and its ability to efficiently target expression of specific genes is well known. But systems that can deliver siRNA in a non-toxic and rapid manner have been lacking. This invention meets this need. It overcomes limitations of current gene delivery systems such as viral based vectors, liposomes and biodegradable polymers using siRNA targeted gene knockdown. Advantages include:
• Rapid release of payload ensures efficient delivery and targeting of genes into specific cells
• The polymer has little to no toxicity
• Stable and enhanced siRNA delivery
Technical Details:
Johns Hopkins researchers have developed methods to synthesize a bioreducible polymer that efficiently and rapidly delivers siRNA into a cell for targeted knockdown of gene expression. This was achieved by including disulfide bonds into backbone of the poly(Beta amino) ester. The disulfide bonds are reduced in the human body by glutathione (GSH) resulting in the rapid and efficient release of the siRNA cargo into the cytoplasm of the cell and leading to the efficient knockdown of target genes. The reducible polymer has the same physical properties as previously developed non-bioreducible polymers but possess a lower toxicity profile. Thus, this invention provides a safe and efficacious siRNA delivery platform for gene therapy targeting various diseases.
Looking for Partners:
To develop & commercialize this technology as siRNA based delivery vehicle platform for gene therapy.
Only Available Non-Exclusively as follows: Non-exclusive for all fields, excluding brain diseases, Facioscapulohumeral muscular dystrophy, Becker muscular dystrophy, Duchenne muscular dystrophy, Dysferlinopathies, Limb-girdle muscular dystrophies, Myotonic dystrophy, Oculopharyngeal muscular dystrophy, X-linked myotubular myopathy, and Usher syndrome.
Stage of Development: Discovery
Data Availability: Human glioblastoma multiforme cell lines
Publications/Associated Cases: Bioreducible Poly (Beta-Amino Ester)s For siRNA Delivery
