Albumin-Binding Polypeptides for Extended Half-Life

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Solution Overview

Problem

Current treatments for metabolic diseases such as lipodystrophy, dyslipidemia, and diabetes lack polypeptides with extended half-lives and convenient dosing regimens, leading to suboptimal therapeutic outcomes.

Innovation Solution

Engineered polypeptides are developed that incorporate an albumin binding domain (ABD) combined with a biologically active hormone domain (HD), such as leptin, to enhance binding affinity for albumin, thereby increasing the duration of action and reducing renal clearance and degradation.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Duration of action of moving object

If conventional polypeptides are used for treating metabolic diseases, then therapeutic efficacy is achieved, but duration of action is short and dosing frequency must be high

Engineering Contradiction:
Improveduration of actionVSAvoiddosing frequency
Core Design Contradiction:
Duration of action of moving objectVSProductivity

Solution Approach 1:

The invention introduces albumin as an intermediary carrier protein that binds to the therapeutic polypeptide. This albumin-polypeptide complex extends the circulation half-life of the therapeutic agent by utilizing albumin's long residence time in plasma, thereby reducing dosing frequency while maintaining therapeutic efficacy

Inventive Principle:
Principle #24Intermediary (Mediator)

Solution Approach 2:

The invention creates a composite therapeutic structure by fusing the active polypeptide with an albumin-binding domain (ABD). This composite molecule combines the therapeutic function of the original polypeptide with the extended circulation properties of albumin, achieving both sustained duration of action and reduced dosing frequency

Inventive Principle:
Principle #40Composite materials

2Reliability

If polypeptides are administered frequently to maintain therapeutic levels, then therapeutic efficacy is maintained, but patient compliance and convenience deteriorate

Engineering Contradiction:
Improvetherapeutic efficacyVSAvoidpatient compliance
Core Design Contradiction:
ReliabilityVSEase of operation

Solution Approach 1:

Albumin serves as a mediator that prolongs the circulation time of the therapeutic polypeptide. By binding to albumin, the polypeptide maintains therapeutic levels for extended periods, enabling less frequent dosing and thereby improving patient compliance without compromising therapeutic efficacy

Inventive Principle:
Principle #24Intermediary (Mediator)

3Duration of action of stationary object

If polypeptides are cleared rapidly by renal filtration, then half-life is short, but therapeutic coverage is insufficient

Engineering Contradiction:
Improvehalf-lifeVSAvoidtherapeutic coverage
Core Design Contradiction:
Duration of action of stationary objectVSReliability

Solution Approach 1:

The invention employs albumin as a protective intermediary that shields the therapeutic polypeptide from rapid renal clearance. The albumin-polypeptide complex is too large for efficient glomerular filtration, thereby extending the half-life and ensuring adequate therapeutic coverage

Inventive Principle:
Principle #24Intermediary (Mediator)

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 engineered polypeptides demonstrate extended duration of action, reduced immunogenicity, and maintained therapeutic efficacy, providing effective treatment for metabolic disorders with improved dosing regimens.

Implementation Method 1

engineered polypeptides which incorporate an albumin binding domain in combination with a biologically active peptide... because the engineered polypeptides described herein can bind albumin, the compounds can be sequestered (e.g., bound to albumin) while in the circulation

Methodology Applied
Scientific EffectAlbumin binding:

Data Source

PatentEP2729160B1Engineered polypeptides having enhanced duration of action and reduced immunogenicity
Publication Date: 2019.03.27 AEGERION PHARMACEUTICALS INC
  • EP2729160B1 patent drawingFigure 1A
  • EP2729160B1 patent drawingFigure 1A
  • EP2729160B1 patent drawingFigure 1A

AI summary

Compounds are provided having inter alia good duration of action, high potency and/or convenient dosing regimens including once weekly administration. The compounds are engineered polypeptides which incorporate an albumin binding domain in combination with one or more biologically active polypeptides. Also provided are pharmaceutical compositions and methods of treatment for diseases and disorders including lipodystrophy, dyslipidemia, hyperlipidemia, overweight, obesity, hypothalamic amenorrhea, Alzheimer's disease, leptin deficiency, fatty liver disease or diabetes (including type I and type II). Additional diseases and disorders which can be treated by the compounds and methods described herein include nonalcoholic steatohepatitis (NASH) and nonalcoholic fatty liver disease (NAFLD), metabolic syndrome X and Huntington's Disease.