CPSA USA 2019

Short Course 1
Protein Identification, Quantification, and Biomarker Analysis

Mass spectrometry, as enabled by modern ionization methods such as MALDI and ESI, has dramatically transformed the science of protein identification and quantification. There is a rapidly growing demand for the quantitative analysis of proteins and peptides by LC-MS/MS in the bio-analytical and clinical laboratories. This short course will help attendees deeply appreciate specific challenges associated with successful outcomes in LC-MS based protein analysis. An in-depth view of the practical aspects involved in protein/peptide analysis by LC-MS/MS will be covered and will include case studies from the literature and from the instructor's personal experiences.

The use of nanospray enabled LC-MS has developed from a qualitative tool for (global) proteomics to a quantitative method suitable for activities such as peptide/protein biomarker validation. Key to success has been the combination of highly specific sample preparation methods, high sensitivity nanospray ionization, and high performance tandem mass spectrometry. Critical parameters involved in robust sample preparation, nanobore LC and nanospray, sensitive & selective MS detection along and their analytical benefits will be emphasized. The transition of the qualitative nLC-MS/MS technology to methods suitable for absolute quantification will be discussed. New tools for cloud based data sharing and the underlying principles for biomarker identification and validation by differential mass spectrometry will be presented. Real world examples will be presented. The course will also feature an additional hands-on "connections workshop" to hone your expertise is making high quality LC-MS fluid connections.

The target audience includes analysts that have been engaged in qualitative proteomics wishing to transition to quantitative methods, analysts that have specialized in small molecule quantification, and those involved in the development of methods for clinical outcomes.

Instructors:
Weixun Wang, Merck
Gary Valaskovic, New Objective
Nalini Sadagopan, Agilent Technologies
Nathan Yates, University of Pittsburgh

Short Course 2
Bioanalysis of Mix Modalities via Hybrid and Orthogonal Approaches

Over the past 10 years, the development of mixed modalities such as therapeutic protein, millia molecule, ADCs, and DNAs have resulted in new bioanalytical and subsequent diagnostic challenges. A number of hybrid or orthogonal approaches have been developed to quantify the different types of biomolecules. This course will give an overview of the methods, practices, technologies and instrumentation used to perform LBA, protein LC-MS, and genomic analysis in a modern bioanalytical laboratory. The course will introduce students to the fundamentals of ligand binding assay, immunogenicity, genomic analysis and orthogonal approaches to improve sensitivity and selectivity of quantification of protein and DNA biomarkers. The courses begin with an introduction to ligand binding assay and the principles of developing a pharmacokinetic (PK) or immunogenicity (IMG) assay, including generation and selection of critical reagents, application of emerging technologies to meet clinical needs. New challenges will also be discussed to align with the newly finalized FDA guidance on PK (2018) and IMG (2019) assay development and validation. The LC-MS based quantitation techniques such as “bottom-up” digestion, pellet digestion, Immunoaffinity Purification will be discussed next with examples covering their usages. The genomic analysis will focus on the impact of next-generation sequencing and its utility in detecting newborn disorders. This course will feature a dynamic format where the latest technologies will be presented and the advantages of hybrid approaches will be highlighted.

Instructors:
Suzanne Cordovado, CDC
Barry Jones, Q2 Solutions
Linlin Luo, Merck
Jim Shen, Bristol-Myers Squibb

Short Course 3
Microsampling and Patient Centric Sampling:
A journey through what it is and how you can incorporate it into your workflows

There is growing interest in the collection and analysis of smaller volume matrix samples (≤100 µL compared to typical traditional volumes ≥200 µL) for the accurate quantitative determination of circulating drug, metabolite and biomarker concentrations in humans and animals. This has been brought about by the increasing sensitivity of analytical systems (particularly LC-MS) and the numerous advantages of implementing these workflows and their associated technologies. These advantages include the ability to collect clinical samples from previously intractable subjects and study locations, i.e. paediatric, critically ill, remote locations, near patient sampling (pharmacists, etc) and at home sampling. In addition, this approach enables additional samples to be collected giving improved data sets, i.e. additional timepoints, analytes, or collection of samples during a clinical event. Further, microsampling facilitates the collection of samples via alternative approaches (viz. finger prick sampling), delivering patient convenience and enables cost savings through ambient temperature sample shipments.

In addition to gaining an understanding of what the drivers for the implementation of microsampling are, attendees of this interactive short course will learn and discuss what techniques are available and will explore the pathways and challenges to the implementation of the approach for their clinical and bioanalytical workflows. This will include addressing the practical, data quality and regulatory issues likely to be encountered. Attendees will understand the benefits and challenges of implementation not only for themselves, but also for other functions and workflows involved in the generation of high quality data for robust decision making and regulatory submission.

Attendance of this short course is encouraged for delegates from a wide variety of backgrounds, including (but not limited to) technology companies with an interest in blood sampling and analysis, clinical trials operations staff, clinical trials experts, pharmacokineticists, regulators and bioanalysts from pharma, biotechs and academia. Delegates will be encouraged to interact with colleagues and will be led by instructors with world leading expertise in the field.

Instructors:
Neil Spooner, Spooner Bioanalytical Solutions
Joe Siple, New Objective
Melanie Andreson, Merck
Kevin Bateman, Merck
Tim Olah, Bristol-Myers Squibb
Enaksha Wickremsinhe, Eli Lilly & Co.

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Short Course 4
Business of Science: Biomarker CRO Selection, Qualification and Oversight

Outsourcing is a common thread across the Biotech and Pharmaceutical industry. Therefore, maintaining solid science, quality and compliance of external providers (CROs) has become a critical element of pharma (i.e. sponsor) responsibilities. Clinical biomarker outsourcing is also part of the outsourcing trend and presents a unique set of challenges. This course and workshop will focus on the selection, qualification and oversight of bioanalytical CROs/vendors that support fit for purpose biomarkers in clinical studies and drug development.

Do you have an understanding of CRO selection process, strategic vendor management, and preferred vendor concepts? We will dive into the need of outsourcing strategies, operational and outsourcing models. We will also discuss CRO selection and benchmarking processes.

Are you confused with terms like GxP, GCLP, and CLIA? Do you want to learn more about risk-based approaches for CRO qualification? How are regulations applied in the context of biomarker use? We will discuss differentiation of exploratory work, biomarkers supporting internal decision-making, biomarkers supporting regulatory decision-making and a tiered approach for CRO qualification.

Are you interested to learn more about vendor oversight? We will discuss a comprehensive (quality management, business, and science) approach to CRO oversight ensuring both compliance and good quality of data produced by CROs. We will use examples for ligand binding and mass spec based assays carried out in CROs to demonstrate the importance of oversight.

The course and workshop will be very interactive and consist of presentations and round table discussions. The target audience includes pharma/biotech (sponsor) colleagues (e.g. science, quality, business or operations functions) involved in outsourcing and collaborating with external partners. The target audience also includes CRO and academics colleagues who are interested to learn more about pharma/biotech requirements and best practices for biomarker outsourcing and becoming preferred partners.

Instructors:
Olga Kavetskaia, Pfizer
Arkady Gusev, Novartis

Short Course 5
Building a Better Clinical Diagnostics / Bioanalytical Mass Spectrometry Lab

Now, more than ever, diagnostics and bioanalytical labs are standalone business entities expected to provide high quality, regulatory robust results quickly while managing profitable business operations in the background on a fee for service basis. This short course digs deep into the operational aspects of producing the results with high quality quickly, so this may be achieved in high quantity per FTE to keep the cost per result decidedly competitive. In addition, this course explores managing the building of the business to find ways to prioritize the right needs at the right time at the right cost. Building a successful bioanalytical lab requires proper infrastructure, acquisition of suitable instruments, recruiting and retaining talented scientists and leadership team. There will be significant emphasis on thorough interaction between all involved so all leave the course knowing compellingly more than they did at the start.

This course focuses on achieving high probability of competitive success in the building and day to day operations of standalone, fee for service clinical diagnostics / bioanalytical mass spectrometry labs. This is addressed in two major areas, 1-highly effective lab operations to bring the deliverables to the customers quickly / efficiently with quality levels that meet / exceed their expectations and 2-building / growing the business to achieve / surpass the goals. The first (lab part) is addressed in discussion of both practical and advanced aspects of the lab science that will drive getting high quality results quickly (LC separations, sample preparation, MS tuning / maintenance, automation, informatics). The second (business part) focuses on the journey from idea to success and it utilizes roundtable discussions / brainstorming sessions to foster creativity. It also includes a successful guest lab builder / owner so that ideas can be put into the context of real world experience.

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Instructors:
Mark Hayward, Active Ingredient Technologies
Ganesh Moorthy, The Children's Hospital of Philadelphia

Guest Discussion Leader:
Shane Needham, Alturas Analytics