Humanized Anti-RANKL Antibody for Osteoporosis Treatment

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Solution Overview

Problem

Current treatments for osteoporosis, such as Prolia, rely on animal-derived monoclonal antibodies that require humanization to reduce immunogenicity, but the process can affect antibody affinity and specificity, and there is a need for more effective monoclonal antibodies that can specifically inhibit the RANKL/RANK pathway to block osteoclast activity.

Innovation Solution

Development of a humanized anti-human RANKL antibody with specific amino acid sequences that can bond effectively with RANKL, inhibiting the RANKL/RANK interaction and osteoclast differentiation, potentially offering improved therapeutic efficacy over existing monoclonal antibodies.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If animal-derived monoclonal antibodies are used to treat osteoporosis, then the antibody can effectively target RANKL and inhibit osteoclast activity, but the antibody induces human anti-animal antibody (HAAA) responses and requires humanization modification

Engineering Contradiction:
Improvetherapeutic effectivenessVSAvoidimmunogenicity
Core Design Contradiction:
ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The patent applies parameter changes by modifying the antibody's amino acid sequence parameters through humanization. The CDR regions from animal antibodies are grafted onto human framework regions, changing the structural parameters to reduce immunogenicity while preserving RANKL binding capability. This is evident in the detailed amino acid sequences provided for the humanized antibody variants.

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The patent uses human framework regions as intermediaries to transfer the antigen-binding function from animal CDR regions. The human framework regions serve as a mediator that allows the animal-derived CDRs to function in a human context without triggering strong HAAA responses, while maintaining the therapeutic effect on RANKL.

Inventive Principle:
Principle #24Intermediary (Mediator)

2Object-affected harmful factors

If humanization modification is performed on animal-derived monoclonal antibodies, then HAAA responses are reduced, but the antibody affinity and specificity may be affected

Engineering Contradiction:
ImproveimmunogenicityVSAvoidantibody affinity
Core Design Contradiction:
Object-affected harmful factorsVSReliability

Solution Approach 1:

The patent applies local quality by preserving the animal-derived CDR regions which contain the local binding specificity for RANKL, while only modifying the framework regions. This localized approach ensures that the critical antigen-binding properties remain intact while reducing immunogenicity in the non-critical framework areas.

Inventive Principle:
Principle #3Local quality

Solution Approach 2:

The patent uses partial humanization where only the framework regions are replaced with human sequences while the CDR regions remain from the original animal antibody. This partial action is sufficient to reduce HAAA responses without completely replacing the sequences that provide RANKL binding affinity and specificity.

Inventive Principle:
Principle #16Partial or excessive action

3Reliability

If the RANKL/RANK signaling pathway is blocked to treat osteoporosis, then bone absorption is inhibited and bone metabolism balance is restored, but the treatment requires highly specific antibodies that currently rely on animal-derived sources

Engineering Contradiction:
Improvebone metabolism regulationVSAvoidantibody production method
Core Design Contradiction:
ReliabilityVSEase of manufacture

Solution Approach 1:

The patent creates humanized copies of animal-derived monoclonal antibodies. The CDR regions which contain the specific recognition code for RANKL are copied from the original animal antibodies and inserted into human framework regions, creating functional copies that maintain therapeutic effectiveness while improving manufacturability for human use.

Inventive Principle:
Principle #26Copying

Applied Scientific Principles

This section explains which scientific principles are used to turn an abstract innovation direction into a practical engineering solution.

Function Achieved in This Case

The humanized anti-human RANKL antibody effectively inhibits osteoclast differentiation and bone absorption, providing a potentially more effective treatment for osteoporosis and other bone loss diseases by specifically targeting the RANKL/RANK pathway.

Implementation Method 1

RANKL is capable of bonding directly with the osteoclast differentiation and activation receptor (abbreviated as ODAR) on the membrane of osteoclast precursors... The humanized anti-human RANKL antibody effectively inhibits osteoclast differentiation and bone absorption, providing a potentially more effective treatment for osteoporosis and other bone loss diseases by specifically targeting the RANKL/RANK pathway.

Methodology Applied
Scientific EffectAntibody-antigen binding:

Data Source

PatentEP3085709B1Humanized Anti-human rankl antibody, pharmaceutical composition and use thereof
Publication Date: 2019.08.21 GENOR BIOPHARMA
  • EP3085709B1 patent drawingFigure 1~3
  • EP3085709B1 patent drawingFigure 4~6
  • EP3085709B1 patent drawingFigure 7~11

AI summary

A humanized the anti-human RANKL antibody, a pharmaceutical composition and a use thereof are provided. The humanized antibody is capable of bonding specifically with human RANKL, a variable region in a heavy chain of the humanized antibody is selected from amino acid sequences of SEQ ID NO: 6, NO: 23, NO: 25, NO: 27 or NO: 29, and a variable region in a light chain of the humanized antibody is selected from amino acid sequences of SEQ ID NO: 14, NO: 31, NO: 33, NO: 35, NO: 37 or NO: 39. Preferably, a complete heavy chain of the humanized antibody is selected from an amino acid sequence of one of SEQ ID NO: 46-50, and/or a complete light chain of the humanized antibody is selected from an amino acid sequence of one of SEQ ID NO: 51-56. The humanized antibody may be used for preparing a medication for the treatment of bone loss diseases.