18248988. NOVEL LIGANDS FOR ASIALOGLYCOPROTEIN RECEPTOR simplified abstract (SANOFI)

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NOVEL LIGANDS FOR ASIALOGLYCOPROTEIN RECEPTOR

Organization Name

SANOFI

Inventor(s)

Bettina Elshorst of Frankfurt am Main (DE)

Gerhard Hessler of Frankfurt am Main (DE)

Armin Hofmeister of Frankfurt am Main (DE)

Ziyu Li of Frankfurt am Main (DE)

[[:Category:Christoph P�verlein of Frankfurt am Main (DE)|Christoph P�verlein of Frankfurt am Main (DE)]][[Category:Christoph P�verlein of Frankfurt am Main (DE)]]

Herman Schreuder of Frankfurt am Main (DE)

Gernot Zech of Frankfurt am Main (DE)

NOVEL LIGANDS FOR ASIALOGLYCOPROTEIN RECEPTOR - A simplified explanation of the abstract

This abstract first appeared for US patent application 18248988 titled 'NOVEL LIGANDS FOR ASIALOGLYCOPROTEIN RECEPTOR

Simplified Explanation

The present disclosure introduces novel ligands that bind specifically to the asialoglycoprotein receptor (ASGPR) and nucleotide analogs conjugated to them for use in oligonucleotides, including double-stranded oligonucleotides like siRNAs.

  • Ligands specifically bind to ASGPR
  • Nucleotide analogs can be incorporated into oligonucleotides
  • Suitable for use in double-stranded oligonucleotides such as siRNAs

Potential Applications

The technology can be applied in targeted drug delivery systems, gene therapy, and molecular biology research.

Problems Solved

This innovation addresses the challenge of specific targeting in drug delivery and gene therapy, enhancing the efficacy and reducing off-target effects.

Benefits

- Enhanced specificity in drug delivery - Improved efficiency in gene therapy - Potential for personalized medicine applications

Potential Commercial Applications

"Targeted Drug Delivery Systems and Gene Therapy Applications Using ASGPR Ligands and Nucleotide Analogs"

Possible Prior Art

There may be prior art related to ligands targeting specific receptors for drug delivery or gene therapy applications, but specific information is not provided in this article.

Unanswered Questions

=== How does this technology compare to existing targeted drug delivery systems? This article does not provide a direct comparison with existing targeted drug delivery systems. Further research or comparative studies would be needed to evaluate the advantages and limitations of this technology in relation to current methods.

=== What are the potential challenges in scaling up the production of these ligands and nucleotide analogs for commercial use? The scalability and cost-effectiveness of producing these novel ligands and nucleotide analogs for large-scale commercial applications are not addressed in this article. Additional studies or pilot projects may be required to assess the feasibility of mass production and commercialization.


Original Abstract Submitted

The present disclosure provides novel piperidine- and guanosine-derived ligands that bind specifically to asialoglycoprotein receptor (ASGPR) and nucleotide analogs conjugated thereto that can be incorporated into oligonucleotides, including double-stranded oligonucleotides such as siRNAs.