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One-step synthesis of cationic steroids for gene delivery, anti-inflammatory activity, and antimicrobial activity.

Description:

Reagents for improved DNA lipofection, depot anti-inflammation, and/or topical antimicrobial therapy

 

Inventor

Scott L. Diamond, Arthur E. Humphrey Professor of Chemical & Biomolecular Engineering

 

 

Technology Overview

Researchers in the Diamond lab have developed a versatile new class of pharmacologically-active molecules with multiple applications.  These molecules deliver plasmids (pDNA), adenovirus (AdV), and adeno-associated virus (AAV) to cultured cells and to pulmonary epithelium in vivo.  In addition to their gene delivery capabilities, the structure of the molecules allows them to function as slow-release prodrugs for local treatment of vector-associated inflammation.  Importantly, this therapeutic effect may synergize with the gene that is delivered.  The anti-inflammatory effect of the cationic steroids does not require DNA, and as the prodrugs degrade, they break down into non-toxic materials with pharmacological activity.  The molecules can be readily synthesized in one-step from inexpensive precursors in a scalable reaction and purified in gram quantities.  Additionally, the cationic steroids have potent antimicrobial activity against gram-positive and gram-negative bacteria, including clinical strains of MRSA and pseudomonas.

 

Caption: Synthesis of a cationic steroid for gene delivery and anti-inflammatory activity.  From Gruneich et al, 2004.

 

Advantages

•       Efficient delivery of genes to cultured cells

•       Synergistic combination of gene delivery and anti-inflammatory effects to enhance gene therapy

•       Rapid production of compound in large quantities

•       Potent and rapid bactericidal activity against drug-resistant bacteria

 

 

Applications

•       Gene therapy via non-viral gene delivery

•       Slow-release therapeutics for treatment of inflammation

•       Enhancer of DNA vaccines

•       Pulmonary infections

 

 

Stage of Development

•       Proof-of-concept and mouse in vivo data 

 

Intellectual Property

USSN 7,442,386

USSN 9,056,048

HK1066485

CA 2,456,977

EPO 1424998

AU 2002324723

 

Reference Media

Gruneich JA et al.  Gene Therapy, 2004, 11(8), p. 668-674.

Gruneich JA and Diamond SL.  J. Gene Med, 2007, 9(5), p. 381-391.

 

 

 

Desired partnerships

• License

Co-development

 

 

Download PDF

 

Docket #  N2612 


Patent Information:
For Information, Contact:
Joshua Jeanson
Associate Director, SEAS/SAS Licensing Group
University of Pennsylvania
jeanson@upenn.edu
Inventors:
Scott Diamond
Keywords: