Phosphane Functionalized Diblock Copolymers for Targeted siRNA Delivery

Award Information
Agency: Department of Health and Human Services
Branch: N/A
Contract: 1R43GM079892-01A1
Agency Tracking Number: GM079892
Amount: $99,857.00
Phase: Phase I
Program: SBIR
Awards Year: 2008
Solicitation Year: 2008
Solicitation Topic Code: N/A
Solicitation Number: PHS2007-2
Small Business Information
DUNS: 148070860
HUBZone Owned: Y
Woman Owned: Y
Socially and Economically Disadvantaged: Y
Principal Investigator
 (651) 209-9757
Business Contact
Phone: (651) 209-9757
Research Institution
DESCRIPTION (provided by applicant): This SBIR project is designed to develop a new non-viral carrier for target-specific delivery of small interfering RNA (siRNA). One of the major challenges in transforming siRNA from laboratory reagents to therapeutics is developing a target-specific delivery system to introduce siRNAs into target cells. Maximal therapeutic benefit with decrease in the required drug dose and avoidance of nonspecific silencing and toxicity in nontarget cells could thereby be achieved. Thi s proposal describes a new diblock copolymer with a positively charged block to complex with siRNA, a poly(ethyleneoxide) block for protection against non-specific adsorption, and a phosphane group on the PEG end for targeting ligand conjugation through St audinger ligation. The Phase II work is expected to investigate the safety and efficacy of this novel siRNA delivery system in an animal model for therapeutic application. Specific aims of this Phase I proposal include: 1) optimize the synthesis of special diblock copolymers; 2) conjugate targeting ligands to diblock copolymers; 3) develop the methodology for preparing siRNA/polymer nanoparticles; 4) evaluate cytotoxicity of the new siRNA delivery system; 5) demonstrate inhibition of gene expression; and 6) investigate intracellular trafficking.

* Information listed above is at the time of submission. *

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