Intra Cellular Delivery of Small Molecules Using Novel Nanotechnology (Case 2060)

Versatile, Robust Nanotube Delivery of siRNA and Nucleic Acid into Cells

The use of small interfering RNA (siRNA), which silences gene expression, holds great clinical promise for the treatment of a variety of diseases. Researchers have been exploring the clinical potential of siRNAs for over two decades, and the first siRNA-based therapeutic, ONPATTRO (patisiran) developed by Alnylam Pharmaceuticals, was finally approved in 2018. The drug development process was extremely hard, with the largest obstacle being how to efficiently and safely deliver siRNAs to desired tissues and cells. There remains an unmet need for a reliable delivery system for transporting siRNA and/or other nucleic acid-based therapeutics, into the cytoplasm. Our invention uses rosette nanotubes to deliver and release siRNA into the cell cytoplasm. 
Market Opportunity
The use of siRNA to silence disease-causing genes holds great clinical promise for the treatment of a variety of diseases, including diabetes, respiratory disease, hepatitis, HIV infection, and cancer. A major advantage of siRNA therapy is its specificity. The greater the specificity, the lower the likelihood of unintended silencing of other genes. The major obstacle to progress using this therapeutic approach, however, is the lack of a safe, reliable delivery system for transporting siRNA into the cytoplasm.
Innovation and Meaningful Advantages
Our invention provides an effective delivery system for transporting siRNA into the cell cytoplasm and opens the way to realizing the clinical potential of the use of siRNA to treat a wide range of diseases. Rosette nanotubes are tubular structures that self-assemble in water and can be tailored to different medical applications. Our methodology involves mixing together rosette nanotubes and one or more nucleic acids under conditions which cause the rosette nanotubes to combine with the nucleic acids and form a stable complex. The use of rosette nanotubes is safe, reliable, and highly specific. Our invention could also have other therapeutic uses based on delivery of DNA or RNA into cells

Principal Investigator
Thomas Webster, PhD
Former Associate Professor of Engineering
and Orthopaedics
Brown University
Brown Tech ID # 2055J

IP Information
Patent Issued 10,364,440; priority date Jan 4, 2011
Patent Issued 10,344,300; priority date Jan 4, 2011

Patent Information:
For Information, Contact:
Brown Technology Innovations
350 Eddy Street - Box 1949
Providence, RI 02903
Thomas Webster
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