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Key Sessions

Sukhanya Jayachandra, Ph.D.

Regulatory Perspective on Analytical Characterization and Bioassay Development for Cell and Gene Therapies

CBER-FDA

Sudhakar Agnihothram, Ph.D.

Product Characterization for Adjuvanted Vaccines

CBER-FDA

Nadine Ritter, Ph.D.

FDA Town Hall

Global Biotech Experts, LLC

7:00am - 8:00am

Coffee & Registration

More
8:00am - 8:05am
Chairperson’s Remarks

Chairperson’s Remarks

More
Showing of Streams
9:45am - 10:10am

Networking Refreshment Break in Poster and Exhibit Hall

More
Showing of Streams
Showing of Streams
12:15pm - 1:25pm

Networking Luncheon in the Exhibit & Poster Hall

More
Showing of Streams
2:30pm - 2:55pm

Networking and Refreshment Break in Poster and Exhibit Hall

More
Showing of Streams
4:30pm - 5:00pm

Panel Discussion: Bioassays for Cell and Gene Therapies - What Kind of Assays are Relevant? What are the Analytical Challenges?

  • Moderator Bhavin Parekh, Ph.D. - Research Advisor, Bioassay Group Leader, BioProduct R&D, Eli Lilly and Co.
  • Panelist Marina Feschenko, Ph.D. - Principal Scientist, Analytical Development- Biologics & Gene Therapy, Biogen
  • Panelist Nancy Sajjadi, MSc. - Consultant, Independent Quality Consultant
  • Panelist Nadine Ritter, Ph.D. - President and Analytical Advisor, Global Biotech Experts, LLC
More
5:00pm - 5:05pm
Close of Conference – See you in 2019!

Close of Conference – See you in 2019!

More
7:00am - 8:00am 60 mins
Coffee & Registration
8:00am - 8:05am 5 mins
Chairperson’s Remarks
8:05am - 8:35am 30 mins
Plenary Session: FDA Perspective – Physical and Functional Characterization
Regulatory Perspective on Analytical Characterization and Bioassay Development for Cell and Gene Therapies
  • FDA Speaker Sukhanya Jayachandra, Ph.D. - OTAT, Division of Cellular and Gene Therapies, CBER-FDA
8:35am - 9:05am 30 mins
Plenary Session: FDA Perspective – Physical and Functional Characterization
Product Characterization for Adjuvanted Vaccines
  • Sudhakar Agnihothram, Ph.D. - Reviewer, CBER-FDA
9:05am - 9:45am 40 mins
Plenary Session: FDA Perspective – Physical and Functional Characterization
FDA Town Hall
  • Moderator Nadine Ritter, Ph.D. - President and Analytical Advisor, Global Biotech Experts, LLC
  • Panelist Lokesh Bhattacharyya, Ph.D. - Lab Chief, CBER - FDA
  • Panelist Alfred Del Grosso, Ph.D. - Team Leader, Analytical Chemistry, Division of FDA/CBER/OCBQ/DBSQC, U.S. FDA
  • Panelist Muhammad Shahabuddin, Ph.D. - Chief, Laboratory of Biochemistry, Virology and Immunology, CBER - FDA
9:45am - 10:10am 25 mins
Networking Refreshment Break in Poster and Exhibit Hall
10:10am - 10:15am 5 mins
Modality Specific Characterization & Strategies for Diverse Products: Stability Studies and Specification Setting
Chairperson's Remarks
  • Y. Diana Liu, MS - Protein Analytical Chemistry, Genentech
10:10am - 10:15am 5 mins
Areas & Applications of Characterization: Upstream/PAT
Chairperson's Remarks
  • John Alvino - Senior Manager, Global RA-CMC, GRAPSQA, Astrazeneca
10:10am - 10:15am 5 mins
Biological Assays: Monitoring Performance, Controls and Reference Management Programs for Bioassays
Chairperson's Remarks
  • Nadine Ritter, Ph.D. - President and Analytical Advisor, Global Biotech Experts, LLC
10:15am - 10:45am 30 mins
Modality Specific Characterization & Strategies for Diverse Products: Stability Studies and Specification Setting
Case Study - Comprehensive Characterization of Charge Variants of Therapeutic Protein Revealing Novel Modification
  • Weitao Jia, Ph.D. - Protein Analytical Chemistry-2, Pharma Technical Development, Genentech Inc
10:15am - 10:45am 30 mins
Info
Areas & Applications of Characterization: Upstream/PAT
Integration of Process Analytical Technology in Upstream Bioprocessing of CHO Cells
  • Erica Fratz-Berilla, Ph. D., - Staff Fellow, FDA CDER

Monitoring of the upstream bioprocess, and ultimately control, is vital to obtain high protein yields and meet all quality specifications for a biotherapeutic. Process analytical technology (PAT) can be used in process development to optimize physical, chemical, and biological parameters that may result in changes in CHO cell growth, titer, and product quality attributes. Additionally, PAT can be used to detect deviations in CHO cell culture parameters that may have otherwise remained unnoticed during the bioprocess. This talk will focus on our use of several PAT tools to monitor and better understand our CHO bioprocess.

10:15am - 10:45am 30 mins
Biological Assays: Monitoring Performance, Controls and Reference Management Programs for Bioassays
Monitoring Performance of Bioassay to Avoid Out of Specification Investigations
  • Emma Reyes, PhD, MBM - Senior Scientist, Pfizer, Inc.
10:45am - 11:15am 30 mins
Info
Modality Specific Characterization & Strategies for Diverse Products: Stability Studies and Specification Setting
Getting to the Same End in Half the Time: Strategies for Efficient Analytical Development from Veterinary Pharma
  • Agatha Feltus - Principal Research Scientist, Pharmaceutical and Vaccine Science & Technology, Elanco, an Eli Lilly Company

Shorter clinical studies in veterinary pharma put development on the critical path to submission. The analytical challenge is to quickly develop, transfer, and validate appropriate stability-indicating methods. Principles of Quality by Design, platform methods, and upfront forced degradation studies are strategies that enable meeting shorter timelines.

10:45am - 11:45am 60 mins
Areas & Applications of Characterization: Upstream/PAT
Process & Analytical Approaches Toward Problem Solving - Case Studies From A Dual Perspective
  • Jimmy Smedley, Ph.D. - Director, Analytical Development, KBI Biopharma, Inc.
  • Yuan Chang, Ph.D. - Director, Downstream Process Development, KBI Biopharma, Inc.
10:45am - 11:15am 30 mins
Biological Assays: Monitoring Performance, Controls and Reference Management Programs for Bioassays
Bioassays as Critical Reagents: Addressing the Needs from Development to Qualification to Long-Term Supply
  • Alpana Prasad, PhD - Product Manager Bioassays, Immuno-oncology, Eurofins DiscoveRx
11:15am - 11:45am 30 mins
Modality Specific Characterization & Strategies for Diverse Products: Stability Studies and Specification Setting
Panel: Why Forced Degradation Studies are Important during Drug Development Process
  • Moderator Santosh Yadav, Ph.D. - Principal Scientist, GVBC-BA, Merck & Company Inc
  • Panelist Huijuan Li, Ph.D. - Head, Analytical Development, Moderna Therapeutics
  • Panelist Chris Rankin, Ph.D. - Associate Director, R&D, MedImmune
11:15am - 11:45am 30 mins
Biological Assays: Monitoring Performance, Controls and Reference Management Programs for Bioassays
Please move to another track
12:15pm - 1:25pm 70 mins
Networking Luncheon in the Exhibit & Poster Hall
1:25pm - 1:30pm 5 mins
Modality Specific Characterization & Strategies for Diverse Products: New Modalities
Chairperson's Remarks
  • Suzanne Hudak, M.S. - Scientist, MedImmune
1:25pm - 1:30pm 5 mins
Areas & Applications of Characterization: Immunogenicity
Chairperson's Remarks
  • Soundara Soundararajan, Ph.D. - Principal Scientist, BioProcess Development-MRL, Merck & Co.
1:25pm - 1:30pm 5 mins
Biological Assays: Phase-Appropriate Method Validation for Biological Assay & Requirements for Quality Compliance
Chairperson's Remarks
1:30pm - 2:00pm 30 mins
Modality Specific Characterization & Strategies for Diverse Products: New Modalities
Analytical Method Overview for Cellular Therapy Drugs
  • Michael Bowen, Ph.D. - Former Head of Analytical Development, Juno Therapeutics, a Celgene Company
1:30pm - 2:00pm 30 mins
Info
Areas & Applications of Characterization: Immunogenicity
Non-Clinical Assessments of Immunogenicity
  • Wojciech Jankowski, Ph.D. - Commissioner's Fellow, CBER - FDA

Immunogenicity (development of anti-drug antibodies) is a significant impediment to development and licensure of any therapeutic-protein. Recent progress in the development and use of non-clinical and pre-clinical assessments of immunogenicity will be presented along with examples demonstrating good predictive outcomes. I will illustrate how judicious application of these tools can permit better decision making during drug-development, licensure, and clinical-trials.

1:30pm - 2:00pm 30 mins
Biological Assays: Phase-Appropriate Method Validation for Biological Assay & Requirements for Quality Compliance
Taking it to Another Level: Phase Appropriate Validation of Bioassays
  • Michael Sadick, Ph.D. - Principal Scientist, Catalent Pharma Solutions
  • Michael Merges - Director of Strategic Growth, Biologics Analytical Services, Catalent Pharma Solutions
2:00pm - 2:30pm 30 mins
Info
Modality Specific Characterization & Strategies for Diverse Products: New Modalities
Characterization of Critical Quality Attributes of mRNA, A Novel Therapeutic
  • Huijuan Li, Ph.D. - Head, Analytical Development, Moderna Therapeutics

Using mRNA to create new therapeutics is complex and requires overcoming novel scientific and technical challenges. mRNA production process heavily relies on in vitro enzymatic synthesis instead of chemical synthesis due to its length. Recent advances towards mRNA production and delivery prompt a need for robust analytical methods capable of characterizing this new class of drugs. mRNA product-related impurities include short mRNAs resulting from either premature termination of transcription or in-process degradation, uncapped mRNAs, and point mutations, insertions/deletions. Multiple case studies utilizing a combination of biochemical and biophysical methods will be discussed on characterization of mRNA product- related impurities and variants for successful development of mRNA therapeutics.

2:00pm - 2:30pm 30 mins
Info
Areas & Applications of Characterization: Immunogenicity
The Critical Need for Mass Spectrometry Characterization of Glycans during Commercial Process Development
  • Barry Drew, Ph.D. - Sr. Scientist II, Bristol-Myers Squibb

Knowledge and the ability to mitigate immunogenicity risks from non-native antigens is critical in the safety and efficacy of biotherapeutics when treating patients. It is imperative that Commercial Process Development (CPD) activities eliminate or reduce the level of immunogenic antigens to a level that is safe for Patients. Some of these risks include the presence of non-human glycoforms, the impact on PK/PD (clearance), FcRn/FcγIII binding (effector function), or glycans that have a measureable impact on potency. From a regulatory perspective, human-compatible and consistent glycosylation are required for a safe and efficacious drug product. This requires that Biologics companies thoroughly characterize the glycosylation profile during Upstream CPD and ensure that any changes observed during development and their impact to the glycosylation profile are well understood before transferring the process to Commercial Manufacturing.


Glycan analysis has historically been carried out by retention time based assays. However, due to previous unknown co-elutions or glycans that did not appear in a standard IgG library, complete assessment of glycans present is difficult or miss assignment of glycans can occur. Changes in media components, feed strategies, temperature shifts, cell line, cell clones, or other upstream changes can have significant impact on glycosylation profiles and the types of glycans being produced. As a result, the use of mass spectrometry in glycan analysis is critical for accurately identifying glycans and better understanding the impact of process conditions to ensure that glycosylation is conserved between First in Human (FIH) and CPD.

2:00pm - 2:30pm 30 mins
Biological Assays: Phase-Appropriate Method Validation for Biological Assay & Requirements for Quality Compliance
Lab Quality and Data Integrity for Non-GMP Labs
  • Nadine Ritter, Ph.D. - President and Analytical Advisor, Global Biotech Experts, LLC
2:30pm - 2:55pm 25 mins
Networking and Refreshment Break in Poster and Exhibit Hall
2:55pm - 3:00pm 5 mins
Modality Specific Characterization & Strategies for Diverse Products: Biosimilar Comparability and Interchangeability
Chairperson's Remarks
  • John Schiel, Ph.D. - Research Chemist, IBBR, National Institute of Standards and Technology
2:55pm - 3:00pm 5 mins
Biological Assays: Development for Challenging Modalities
Chairperson's Remarks
3:00pm - 3:30pm 30 mins
Modality Specific Characterization & Strategies for Diverse Products: Biosimilar Comparability and Interchangeability
FDA Draft Guidance on Interchangeability - Industry Perspective
  • Hillel Cohen - Executive Director, Scientific Affairs, Sandoz Inc.
3:00pm - 3:30pm 30 mins
Biological Assays: Development for Challenging Modalities
Bioassays for a Bispecific Adntibody Drug Conjugate
3:30pm - 4:00pm 30 mins
Info
Modality Specific Characterization & Strategies for Diverse Products: Biosimilar Comparability and Interchangeability
The Role of Higher Order Structure in the Assessment of Comparability
  • Mats Wikström, Ph.D. - Principal Scientist, Group Leader, Higher Order Structure, Amgen

Improvements in production methods and process and control test methods along with the development of highly sensitive analytical techniques for product characterization have helped to evolve the regulation of biological products by allowing manufacturers to readily identify and assess the impact of manufacturing changes on the product. Key to these methods are cutting edge and highly sensitive techniques to assess differences in higher order structure (HOS). In many cases, sensitive analytical and functional characterization can be paired with prior knowledge of the process and product to demonstrate product comparability between a pre- and post-change biologic through a comparability exercise, many times without the need for additional clinical data. However, analytical/functional characterization alone cannot be relied upon to establish clinical equivalence after major manufacturing changes. While techniques to probe higher order structure have advanced significantly, in some cases, the relationship between small, but detectable, structural differences and the safety, efficacy, and immunogenicity of a biological product cannot yet be readily established and thus may require additional clinical data. Therefore, while the assessment of higher order structure is highly important in the establishment of comparability, the degree to which HOS data obtained by currently available methods can be relied upon in these exercises is distinctly different.

3:30pm - 4:00pm 30 mins
Biological Assays: Development for Challenging Modalities
Comparability Assessment Using SPR Method and How to Avoid the Pitfalls Associated with the Increased Precision of the Method Compared to Other Bioassays
  • Jeongsup Shim, Ph.D. - Scientist, Analytical Development and Quality Control - Biological Technologies, Genentech
4:00pm - 4:30pm 30 mins
Info
Modality Specific Characterization & Strategies for Diverse Products: Biosimilar Comparability and Interchangeability
Understanding Deficiencies Identified During the Biosimilarity Exercise
  • Nitisha Pyndiah, Ph.D. - Consultant, HartmannWillner

For a product to be demonstrated to be biosimilar to a reference product it must be highly similar and have no clinically meaningful differences but when are differences considered not highly similar and clinically meaningful? How do you proceed when biosimilarity is not demonstrated? In this presentation, we aim at providing ample information about challenges associated with the demonstration of biosimilarity. We propose to present two case studies of monoclonal antibody biosimilars to highlight deficiencies identified during the biosimilarity exercise. From analytical data with differences in charge variants and purity to potency data and manufacturing changes, targeting the most common deficiencies regarding biosimilarity will help your product reach patients faster. FDA considers the totality of evidence approach when evaluating biosimilars and sponsors/applicants are advised to make the most of this approach.

4:00pm - 4:30pm 30 mins
Info
Biological Assays: Development for Challenging Modalities
How Far Should we go to Demonstrate Potency of Gene Therapy Drugs In Vitro?
  • Marina Feschenko, Ph.D. - Principal Scientist, Analytical Development- Biologics & Gene Therapy, Biogen

Potency of the drug is the most critical quality attribute and needs to be assessed for lot release and stability. The potency assays have to be reflective of the mechanism of action, specific, and quantitative. These assays are usually more difficult to develop and not possible to share between different programs. Potency of gene therapy drugs can be assessed in animal models and cell-based assays. The lack of relevant animal models for human diseases coupled with large variability and high cost of testing makes them a rare choice for drug release. The cell-based assays can measure three different activities of the viral vector: ability to penetrate cells (infectivity), target protein expression, and functional potency. Do we need to have all three or we can justify selecting one or two assays for release? Our approach for two different AAV-based programs will be presented and discussed.

4:30pm - 5:00pm 30 mins
Panel Discussion: Bioassays for Cell and Gene Therapies - What Kind of Assays are Relevant? What are the Analytical Challenges?
  • Moderator Bhavin Parekh, Ph.D. - Research Advisor, Bioassay Group Leader, BioProduct R&D, Eli Lilly and Co.
  • Panelist Marina Feschenko, Ph.D. - Principal Scientist, Analytical Development- Biologics & Gene Therapy, Biogen
  • Panelist Nancy Sajjadi, MSc. - Consultant, Independent Quality Consultant
  • Panelist Nadine Ritter, Ph.D. - President and Analytical Advisor, Global Biotech Experts, LLC
5:00pm - 5:05pm 5 mins
Close of Conference – See you in 2019!