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

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

 

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.
 

Inventor

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

 

 

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: 

Reference Media: 

Desired Partnerships: 

  • License
  • Co-development

Docket # N2612 

 

 

Download PDF

 


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