Nanobody Formulation Stability via Buffer and Salt Control

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Solution Overview

Problem

Current formulations of Nanobodies face challenges with stability and solubility, leading to aggregation, degradation, and loss of biological activity during storage and transportation due to chemical and physical instabilities, necessitating the development of stable and soluble formulations for effective administration.

Innovation Solution

The formulation includes an aqueous carrier with a pH of 5.5 to 8.0, a polypeptide concentration of 120 mg/mL or more, a buffer concentration of 10mM to 100mM histidine pH 6.0-6.5, and an inorganic salt concentration of 150 mM or less, along with excipients like saccharides and surfactants to enhance solubility and stability, thereby maintaining the integrity of single variable domains under various stress conditions.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If Nanobodies are formulated in conventional liquid preparations, then they can be administered to patients, but they exhibit short shelf lives and loss of biological activity due to chemical and physical instabilities during transportation and storage

Engineering Contradiction:
Improvestability of NanobodyVSAvoidshelf life
Core Design Contradiction:
ReliabilityVSDuration of action of stationary object

Solution Approach 1:

The patent optimizes formulation parameters including pH (5.5-8.0), ionic strength (150 mM or lower), and component concentrations (buffer 10-100 mM, excipients 0.1-5%, surfactants 0.01-1%) to achieve stable liquid formulations with extended shelf life and minimal loss of biological activity under various storage conditions

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The patent introduces excipients and surfactants as intermediary substances that mediate between the Nanobody and the storage environment, protecting the Nanobody from chemical degradation (deamidation, oxidation) and physical instability (aggregation, precipitation) during transportation and storage

Inventive Principle:
Principle #24Intermediary (Mediator)

2Productivity

If Nanobodies are concentrated to high levels to reduce administration volume, then dosing efficiency improves, but solubility and aggregation issues arise

Engineering Contradiction:
Improvedosing efficiencyVSAvoidsolubility and aggregation
Core Design Contradiction:
ProductivityVSStability of the object's composition

Solution Approach 1:

The patent adjusts formulation parameters including pH, ionic strength, and adds specific excipients and surfactants to enable stable formulations at high Nanobody concentrations (120 mg/mL or more), achieving both dosing efficiency and solubility

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The patent creates composite formulations combining Nanobodies with multiple components (buffers, excipients, surfactants) that work synergistically to maintain solubility and prevent aggregation at high concentrations, enabling concentrated dosing without compromising stability

Inventive Principle:
Principle #40Composite materials

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 formulation achieves high solubility and stability, with less than 10% aggregation and pyroglutamate formation, retaining over 80% binding activity after storage under stress conditions, including extreme temperatures and mechanical stress, ensuring prolonged potency and efficacy.

Implementation Method 1

an aqueous carrier with a pH of 5.5 to 8.0 and a polypeptide comprising one or more single variable domains at a concentration of 120 mg/mL or more

Methodology Applied
Scientific EffectBuffering:

Implementation Method 2

the presence in the formulation of certain buffers, certain excipients and/or certain surfactants may increase the solubility, the melting temperature and/or the stability of the single variable domains

Methodology Applied
Scientific EffectSurfactant action: Surfactant

Implementation Method 3

Chemical instability may be caused by deamidation, racemization, hydrolysis, oxidation, beta elimination or disulfide exchange

Methodology Applied
Scientific EffectChemical stabilization: Oxidation

Implementation Method 4

The formulation includes an aqueous carrier with a pH of 5.5 to 8.0, a polypeptide concentration of 120 mg/mL or more, a buffer concentration of 10mM to 100mM histidine pH 6.0-6.5, and an inorganic salt concentration of 150 mM or less

Methodology Applied
Scientific EffectSolubility enhancement: Solvation

Data Source

PatentEP3438126B1Stable formulations of polypeptides and uses thereof
Publication Date: 2020.08.19 ABLYNX NV
  • EP3438126B1 patent drawingFigure 1(A)
  • EP3438126B1 patent drawingFigure 1(B)
  • EP3438126B1 patent drawingFigure 2

AI summary

Formulations are provided that contain single variable domains with a good solubility and good stability under different storage, transportation and stress conditions. The formulations are useful as pharmaceutical formulation. The formulation comprises an aqueous carrier with a pH of 5.5 to 8.0, a buffer selected from the group consisting of histidine pH 6.0-6.5, hepes pH 7.0-8.0, MES pH 6.0, succinate pH 6.0-6.5 and acetate pH 5.5-6.0; an excipient; and/or a surfactant selected from Tween 80, Tween 20 and poloxamers. The formulation is further characterized that it has an inorganic salt concentration of 150 mM or lower. The invention further relates to containers and pharmaceutical units comprising such formulations and to methods for preparing and prophylactic and therapeutic uses of the formulations and pharmaceutical units of the invention.