2016 Archived Content

Cambridge Healthtech Institute’s 3rd Annual
Protein Aggregates & Particles

Understanding Aggregation Kinetics to Effect Control and Minimise Immunogenicity Risks

3 - 4 November 2016 | EPIC SANA Lisboa Hotel | Lisboa PORTUGAL


Aggregation impacts biopharmaceutical development at every stage from discovery to development to processing. The mechanisms and kinetics of aggregation, particularly of the novel and complex biomolecules, are not well-defined, thus regulators are highly concerned about its impact on drug product safety and immunogenicity. As such, companies are expending all efforts to predict, identify, characterize, control and minimize the occurrence of aggregates and particles.

The 3rd Annual Protein Aggregates & Particles conference will bring together protein chemists and biochemists, analytical scientists and engineers to uncover the underlying mechanisms of aggregation, predict aggregation propensity using in silico and modeling tools, design and engineer novel biomolecules with reduced aggregation, as well as formulate drug products to minimize aggregates and particulates.

Final Agenda

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Recommended Short Course*

SC8: Protein Aggregation: Mechanism, Characterisation and Consequences

*Separate registration required


THURSDAY 3 NOVEMBER

12:30 Registration

13:00 Dessert Break in the Exhibit Hall with Poster Viewing


AGGREGATION AND IMMUNOGENICITY RISKS

13:30 Chairperson’s Opening Remarks

Jeremy Derrick, Ph.D., Professor, Molecular Microbiology, University of Manchester


13:35 KEYNOTE PRESENTATION:
The Immunogenicity of Antibody Therapeutics:
Aggregates and Immune Complexes

Roy_JefferisRoy Jefferis, Ph.D., MRCP, FRCPAth, D.Sc., Professor Emeritus, University of Birmingham

Many parameters contribute to the development of anti-drug antibodies (ADA) to protein therapeutics; aggregation being considered a particular risk. However, administration of IgG antibody therapeutics results in the formation immune complexes (aggregates!) that may similarly activate pathways leading to ADA production. Individual mAb therapeutics should be evaluated for the propensity of both aggregates and immune complexes to activate downstream effector cascades.


14:20 Understanding the Relationship between Aggregation and Immunogenicity of Biotherapeutic Proteins

Jeremy_DerrickJeremy Derrick, Ph.D., Professor, Molecular Microbiology, University of Manchester

Aggregation is known to play an important part in the immunogenicity of therapeutic proteins. I will describe recent experiments which compared the immunological responses to immunization of selected example proteins, in monomeric and aggregated forms, in a mouse model. In addition, we also examined the effect of the heat shock protein DnaK, a common HCP, and show that it can play a role in modulating the immune response to aggregates.

14:50 Formulate and Characterize More Biologic Formulations than Ever before

Daniel Lund, Ph.D., Applications Scientist, Unchained Labs

Biologic formulations are hard to make and difficult to characterize. The process to buffer exchange and concentrate proteins can take days. Screening them for stability take days to weeks and figuring out if they will aggregate can take months. In this talk, we’ll present a rapid, total solution for formulating proteins, characterizing them for stability, predicting whether they will aggregate and determining the pathway of aggregation.

15:20 Refreshment Break in the Exhibit Hall with Poster Viewing

16:05 Using Denaturant Solutions to Probe the Colloidal Stability of Partially Folded Proteins and the Link to Aggregation

Robin Curtis, Ph.D., Senior Lecturer, School of Chemical Engineering and Analytical Sciences, University of Manchester

16:35 Immunogenicity Risk of Protein Aggregates – Could Bedside Filtration Be of Help?

Gerhard_WinterGerhard Winter, Ph.D., Professor, Pharmaceutical Technology and Biopharmaceutics, University of Munich

Characterization of protein particles has become a regular request by the authorities and measures to reduce such contaminations are taken. But, it will remain impossible to guarantee for each single container the absence of particles. Why has bedside filtration not been applied more often to reduce the potential risk dramatically? We will present the status quo of bedside filtration of biopharmaceuticals and describe technical aspects like injection forces, particle shedding, particle removal, dead volumes.

17:05 End of Day

17:00 Dinner Short Course Registration


Recommended Dinner Short Course*

SC8: Protein Aggregation: Mechanism, Characterisation and Consequences

*Separate registration required


Day 1 | Day 2 | Speaker Biographies | Download Brochure

FRIDAY 4 NOVEMBER

08:00 Registration and Morning Coffee


MECHANISMS AND KINETICS OF AGGREGATION

08:30 Chairperson’s Remarks

Hans Kiefer, Ph.D., Professor, Applied Biotechnology, Biberach University of Applied Sciences


08:35 KEYNOTE PRESENTATION:
Impact of Non-Proteinaceous Particles on the Characterization and Formation of Protein Particles

Wim_JiskootWim Jiskoot, Ph.D., Professor, Gorlaeus Laboratories, Division of Drug Delivery Technology, Leiden Academic Centre for Drug Research (LACDR)

Particulate impurities in protein formulations pose an analytical challenge and may compromise protein stability. In this presentation I will present a case study about the root cause of nanoparticulate (100C200 nm) impurities in protein formulations containing commonly used sugar excipients. These impurities interfere with the characterization of proteinaceous particles by dynamic light scattering and nanoparticle tracking analysis. Moreover, they compromise protein stability and potentially increase their immunogenicity.


09:05 Mechanisms of Protein Aggregation

Thomas_LaueThomas Laue, Ph.D., Professor, Molecular, Cellular & Biomedical Sciences, University of New Hampshire

The proximity energy framework will be described as a useful way to think about high concentration solutions. Protein solvation, hydrophobic interactions and molecular crowding are incorporated into this framework. Protein charge contributes significantly to preventing aggregation and reducing viscosity. Charge cannot be calculated, so the importance and simplicity of measuring charge will be emphasized. Data presented showing how proximity energies impact protein-protein interactions in high concentration formulations as well as in serum.

09:35 Elucidation of Aggregation Mechanisms in Therapeutic Protein, and Prediction of Formulation Strategies

Paul_DalbyPaul Dalby, Ph.D., Professor, Biochemical Engineering, University College London

Various biophysical methods have investigated the aggregation kinetics, solution conformations, and conformational stabilities of several IgGs and Fab molecules. Structural changes in these molecules have been observed that correlate with their aggregation propensities. These studies have been complemented by molecular dynamics and docking simulations that then together reveal the potential features of protein structure that promote aggregation propensity, and also the potential mechanisms by which particular excipients increase protein stability.

10:05 Coffee Break in the Foyer with Poster Viewing


EVALUATING AGGREGATION PROPENSITY

10:35 Relationship between Aggregate Propensity and Biophysical Parameters of Proteins in Solution

Susumu_UchiyamaSusumu Uchiyama, Ph.D., Associate Professor, Graduate School of Engineering, Osaka University

Aggregates formation of therapeutic proteins is one of the issues that should be solved. Here I will show how experimentally obtained biophysical parameters of proteins in solution, such as secondary virial coefficient and thermal unfolding temperature, are useful to predict aggregates formation during transport and storage. In addition, how chemical modification of protein changes its stability will be introduced. Finally total formulation developments of therapeutic proteins in solution are proposed.

11:05 Large Scale All-Atom Molecular Dynamics Analysis of Multi-Peptide Systems Reproduces Peptide Aggregation Propensity In Line with Experimental Observations

Yutaka_KurodaYutaka Kuroda, Ph.D., Associate Professor, Life Science and Biotechnology, Tokyo University of Agriculture and Technology

We carried out all-atom molecular dynamics simulation for 18 systems containing ~3x104 water molecules and 27 tetra-peptides made from a single amino acid type and using a standard force-field without “artificial” hydrophobic forces. Surprisingly, hydrophobic peptides rapidly formed clusters whereas hydrophilic ones remained monomeric in line with experimental expectations. We believe that this study represents a step toward a molecular understanding of peptide/protein solubility, based on physico-chemical first principles.


DEVELOPING PARTICLE STANDARDS

11:35 Development and Applications of Protein-Like Reference Materials for Monitoring Protein Particles

Srivalli Telikepalli, Ph.D., Research Chemist, National Institute of Standards and Technology

NIST is developing subvisible and visible particle standards using ethylene tetrafluoroethylene polymer and SU-8 photoresist since these materials better mimic the morphology and optical properties of protein particles compared to the currently available particle standards. The development, stability, and challenges associated with working with these materials will be briefly described. Applications of the ETFE particles to standardize subvisible particle measurements and to standardize visual inspection processes will be discussed.

12:05 When Standard Formulation Strategies Fail - Recombinant Albumin for Stabilization of Hard-to-Formulate Biotherapeutics

Phil_MortonPhil Morton, Ph.D., Science Director, Bioprocess & Characterisation, Albumedix Ltd.

The expanding field of biotherapeutics gives promise for improvement of several treatment options. Many of the biopharmaceuticals found to be efficacious, however, continue to face ex vivo instability challenges that are not readily solved by standard excipients. Recombinant human albumin, however, can potentially alleviate these shortcomings. The mechanisms by which albumin help stabilize biopharmaceuticals are multiple and dependent on the specific drug. Data is presented here that exemplifies these different mechanisms.

12:35 Problem-Solving Breakout Discussions with a Light Snack in the Foyer*

Methods and Techniques for Particle Analysis and Characterization

moderator: Jonas Hoeg Thygesen, Ph.D., Research Scientist, R&D, Microanalysis Centre, Novo Nordisk Pharmatech A/S

  • Flow microscopy (ie MFI or FlowCam etc), may be seen as the de facto industry standard for subvisible particle characdterization, but what are the alternatives?
  • What are the strengths and weaknesses of the current analytical methods for particle identification and characterization?
  • How should industry react to USP <787>?
  • What do you see as the future methods within particle analysis?

Aggregation and Viscosity: Understanding Protein-Protein Interactions at High Concentrations

moderator: Thomas Laue, Ph.d., Professor, Molecular, Cellular and Biomedical Sciences, University of New Hampshire

  • Solubility and viscosity are properties of both the protein and the solvent. What properties are important, and what control do you have over them?
  • The proximity energy framework provides a useful means to understand the underlying mechanisms of protein-protein interactions
  • This framework also is useful for the rational design of solvents
 

13:35 Session Break


PARTICLE ANALYSIS AND CHARACTERISATION – DETECTION, IDENTIFICATION, CHARACTERISATION AND CONTROL

14:00 Chairperson’s Remarks

Thomas Laue, Ph.D., Professor, Molecular, Cellular & Biomedical Sciences, University of New Hampshire

14:05 Comparisons between Particle Identification Techniques

Jonas Hoeg Thygesen, Ph.D., Research Scientist, R&D, Microanalysis Centre, Novo Nordisk Pharmatech A/S

MicroFlow Imaging (MFI), Raman and Fourier-Transform Infrared (FTIR) Spectroscopy, Electron- and Optical microscopy are strong and versatile tools for particle identification and characterization. This talk will provide specific cases to highlight advantages and challenges using the different techniques, and furthermore, illustrate how they may complement each other.

14:35 Microscopy-Based Particle Counting and Characterization in a High-Throughput Formulation Screening Platform

Christian_RiedChristian Ried, Ph.D., Senior Scientist, Drug Product Development, AbbVie Deutschland GmbH & Co. KG

Protein particles formed from biologics during manufacture and storage might trigger unwanted effects like anti-drug antibody response in patients. Recently, we have set up an automated platform for high-throughput formulation screening of biologics. Here we present a method that allows for particle counting and characterization from microliter-sized samples as a final step in the screening process.

15:05 Mitigation of Aggregation during Elution from Protein A

John K. Kawooya, Ph.D., Director, Biologics Optimization, Amgen, Inc.

Bispecific protein engineering has produced many new “antibody-like” molecules that are not as stable as antibodies. Some of these molecules aggregate upon exposure to the strong acidic pH (2.5-3.7) encountered during elution from Protein Affinity columns. Presented here are strategies for mitigating aggregation by either protecting the molecules at low pH or by eluting the molecules at neutral pH (7.2) or mildly acidic pH (4.5-5.2).

15:35 Identification and Discriminant Analysis of Subvisible (Proteinaceous and Non-Proteinaceous) Particles

Zahir_AkhunzadaZahir Akhunzada, Ph.D., Research Scientist, PPD, Bristol-Myers Squibb

This presentation discusses a method that successfully characterizes and distinguishes, both potentially proteinaceous and non-proteinaceous subvisible particles (SVPs) in protein formulations by using Microflow Imaging in conjunction with the MVAS software. Discriminant analysis approach will be discussed that will include measurements on seven morphological and light intensity parameters: Image Shape ratio, (C-circularity); Size; Pixel Intensity. The goal is to identify a mechanism that will discriminate a particle based on its parametric measurements.

16:05 QSAR Analysis of Additive Effects on the Aggregation of Monoclonal Antibodies in Downstream Processing

Hans_KieferHans Kiefer, Ph.D., Professor, Applied Biotechnology, Biberach University of Applied Sciences

We have established model experiments to induce aggregation of monoclonal antibodies through various stresses common in downstream processing. Aggregation rate constants of nucleation and growth were extracted from kinetic traces. In an additive screen using 150 compounds, molecular descriptors were correlated with rate constants and thermal stability changes using a quantitative structure activity relationship (QSAR) approach. Molecular properties of additives correlating with protective effects were extracted.

16:35 From in silico Simulations to Market: Improving Antibody CMC Protein Aggregation Properties by Rational Design

Patrick Garidel, Ph.D., Head, Protein Science, Boehringer Ingelheim Pharma GmbH & Co. KG

Early detection and mitigation of CMC risk factors such as protein aggregation propensity is vital for predictable and short timelines in the development of biologicals. We assembled a set of bioinformatics methods to assess candidate amino acid sequences for thermodynamic and colloidal stability and complementary biophysical methods to link a subset of residues to these properties. This knowledge serves as a basis for protein engineering to enhance developability.

17:05 End of Conference



Day 1 | Day 2 | Speaker Biographies | Download Brochure