18359569. LDL RECEPTOR-DIRECTED BISPECIFIC BINDING AGENT-LIGAND FUSIONS FOR THE DEGRADATION OF TARGET PROTEINS simplified abstract (THE REGENTS OF THE UNIVERSITY OF CALIFORNIA)

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LDL RECEPTOR-DIRECTED BISPECIFIC BINDING AGENT-LIGAND FUSIONS FOR THE DEGRADATION OF TARGET PROTEINS

Organization Name

THE REGENTS OF THE UNIVERSITY OF CALIFORNIA

Inventor(s)

Katarina Pance of San Francisco CA (US)

James A. Wells of San Francisco CA (US)

LDL RECEPTOR-DIRECTED BISPECIFIC BINDING AGENT-LIGAND FUSIONS FOR THE DEGRADATION OF TARGET PROTEINS - A simplified explanation of the abstract

This abstract first appeared for US patent application 18359569 titled 'LDL RECEPTOR-DIRECTED BISPECIFIC BINDING AGENT-LIGAND FUSIONS FOR THE DEGRADATION OF TARGET PROTEINS

Simplified Explanation

The present disclosure relates to targeted degradation platform technology, specifically bispecific binding agents for degrading endogenous proteins using the lysosome pathway.

  • Bispecific binding agents for degrading endogenous proteins
  • Utilizing the lysosome pathway for protein degradation
  • Methods for producing such agents
  • Nucleic acids encoding the bispecific binding agents
  • Genetically modified host cells with the nucleic acids
  • Methods for modulating cell activity and treating disorders

Potential Applications

The technology can be applied in the fields of targeted protein degradation, drug development, and gene therapy.

Problems Solved

This technology addresses the challenge of selectively degrading specific endogenous proteins within cells, which can be crucial for treating various diseases.

Benefits

The technology offers a precise and efficient method for degrading target proteins, potentially leading to more effective treatments for a range of disorders.

Potential Commercial Applications

Potential commercial applications include pharmaceuticals, biotechnology, and research tools for protein degradation studies.

Possible Prior Art

Prior art may include other methods of targeted protein degradation, such as PROTACs or molecular glues, but the specific use of bispecific binding agents for lysosome pathway degradation may be novel.

Unanswered Questions

How does this technology compare to other targeted protein degradation approaches on the market?

This technology offers a unique approach using bispecific binding agents and the lysosome pathway for protein degradation, which may have advantages over existing methods in terms of specificity and efficiency.

What are the potential limitations or challenges in implementing this technology in clinical settings?

One potential challenge could be the delivery of the bispecific binding agents to target cells in a controlled and effective manner. Additionally, the safety and efficacy of this approach in human patients would need to be thoroughly evaluated in clinical trials.


Original Abstract Submitted

The present disclosure relates to targeted degradation platform technology. For example, the present disclosure relates to bispecific binding agents for degrading endogenous proteins, whether membrane-associated or soluble, using the lysosome pathway. The disclosure also provides methods useful for producing such agents, nucleic acids encoding same, host cells genetically modified with the nucleic acids, as well as methods for modulating an activity of a cell and/or for the treatment of various disorders.