Description:
- Therapeutic delivery
- Biomedical research
- Drug development
Abstract
USC researchers have generated PAR polymers with high therapeutic potential. They synthesized PAR polymers conjugated with human granulocyte colony-stimulating factor (GCSF) protein using auto-poly- ADP-ribosylation. The resulting PAR polymer-based conjugate, with multivalent GCSF ligands, demonstrates potent cell proliferative activity. In animal studies, mice treated with a single dose of the PAR polymer-GCSF conjugate showed sustained high levels of neutrophils in their blood for 11 days, indicating excellent in vivo efficacy. This innovative method positions functionalized PAR polymers as a promising platform for enhancing the pharmacological activities of therapeutic proteins or peptides.
Benefit
• Promising new approach for therapeutic delivery
• Capable of conjugating with human GCSF protein
• PARylated PARP1-GCSF conjugate stimulates cell proliferation effectively
Market Application
Poly-ADP-ribose (PAR), a naturally occurring polymer, holds potential as a safe and effective therapeutic delivery system. PAR regulates important cellular pathways and can modify proteins through covalent attachments of ADP-ribose moieties. Despite extensive research on PARylation, its therapeutic applications remain unexplored, but PAR's high hydrophilicity and biocompatibility make it a promising drug carrier. Additionally, conjugating therapeutic proteins with PAR may enhance their efficacy due to increased valency. This presents an untapped market opportunity for developing PAR-based drug delivery systems.
Publications
Cheng et al., 2022: A Poly-ADP-Ribose Polymer–GCSF Conjugate
Other
Stage of Development
• Conjugated PAR polymers successfully synthesized
• Tested in vivo in mice
• Available for licensing
Intellectual Property Status
Pending US provisional