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WARF: P06346US

Elastin-Like Biopolymers as Delivery Vehicles for Gene Therapy


INVENTORS -

Darin Furgeson, Younsoo Bae

The Wisconsin Alumni Research Foundation (WARF) is seeking commercial partners interested in developing intelligent biosynthetic nanobiomaterials (IBNs) that serve as non-viral, highly biocompatible and actively targeted delivery vehicles.
OVERVIEWIntelligent biosynthetic nanobiomaterials (IBNs) represent the next evolutionary advance in macromolecular drug delivery—genetically engineered biopolymers.  In contrast to traditional polymer macromolecules or viral carriers used for gene and drug delivery, these biopolymers are non-immunogenic and monodisperse, and offer unparalleled control of structural and biophysical characteristics.

Designed using an mRNA template, protein-based IBNs contain a polycationic region, a thermosensitive elastin-like polypeptide (ELP) sequence, and peptides that target the biopolymer to a specific cell or tissue.  Elastin-like polypeptides have a useful thermal property—they rapidly and reversibly transition from elongated chains to tightly folded aggregates at a pre-determined temperature.  IBNs containing an ELP core can be readily expressed (> 200 mg/L) and purified from E. coli cultures by exploiting this thermosensitivity.  The thermal properties of ELPs also provide an additional means of targeting IBNs to a specific area of the body via hyperthermia.
THE INVENTIONUW-Madison researchers have developed IBNs capable of actively targeting therapeutic nucleic acids, or other negatively charged molecules, to specific cells or tissues.  Positively charged amino acid residues that make up the polycationic region within the IBN platform electrostatically condense the negatively charged, polyanionic nucleic acids into an electroneutral core, where they are protected from degradation.  Targeting modalities, such as ligands, monoclonal antibodies or single chain variable fragment antibodies, are linked to this core by the ELP sequence.
APPLICATIONS
  • Gene therapy
  • Protein, peptide or drug delivery
  • Protein purification
KEY BENEFITS
  • Provides a non-viral, highly biocompatible and actively targeted delivery vehicle
  • Production is easily scalable.
  • Molecular weight and architecture of the IBNs can be precisely controlled.
  • ELP-based IBNs are naturally non-immunogenic.
  • Nucleic acid cargo, such as siRNA, is fully protected from degradation until it reaches the drop point.
  • ELP sequence provides particle stability, favorable pharmacokinetics, and potential for additional hyperthermic targeting.
  • mRNA template design allows for facile modification of the polycationic and ELP regions, and enables insertion of multiple targeting domains.
ADDITIONAL INFORMATION
Contact Information
For current licensing status, please contact Andy DeTienne at adetienne@warf.org or 608-960-9857.
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