WELCOME TO GENE THERAPY ANALYTICAL DEVELOPMENT 2020

8:00 am Chair’s Opening Remarks

8:15 am Submitting a Confident Gene Therapy Analytical Package: Approved Product

  • Fengrong Zuo Senior Director, Analytical Development Department, CMC Regulatory, Quality & Analytical Development , AveXis

Synopsis

• Highlighting analytical challenges comparing therapeutic antibodies/recombinant proteins to the new AAV-based gene therapy modality

• Discussing the analytical strategy toward global regulatory filings and approvals of Novartis’ ZOLGENSMA

• Sharing lessons learned: case studies

8:45 am Addressing Critical Quality Attributes: Analytical Development Support for the Implementation of Gene Therapy Platforms

  • Joseph Lee Executive Director, Analytical Development, Gene Therapy , PTC Therapeutics

Synopsis

• Introduction/overview of the regulatory environment for gene therapies

• Establishing critical quality attributes for gene therapies

• Technical descriptions of assays addressing CQAs

• Additional considerations for gene therapy applications

9:15 am Session Reserved for BIA Separations

9:45 am Panel Discussion: Gene Therapy Analytical Challenges: What to Expect

  • Fengrong Zuo Senior Director, Analytical Development Department, CMC Regulatory, Quality & Analytical Development , AveXis
  • Mark Galbraith Head of Quality Control & Analytical Sciences, Spark Therapeutics
  • Elizabeth Higgins Director, Analytical Development, Voyager Therapeutics
  • Joseph Lee Executive Director, Analytical Development, Gene Therapy , PTC Therapeutics

Synopsis

• Hear from advanced companies in the gene therapy landscape to discuss their most pressing challenges

• Listen to the advice and lessons learned from later stage developers

• Gain insights into future suggestions to improve the analytical process and regulatory feedback loop

10:30 am Speed Networking & Morning Refreshments

Synopsis

Speed networking is the ideal opportunity to get face-to-face time with many of the brightest minds working in gene therapy. Benchmark against the industry leaders and establish meaningful business relationships to pursue for the rest of the conference and beyond.

BIOASSAYS

Overcoming Potency Assay Challenges

MOLECULAR BIOLOGY

Improving Technologies For Detecting Impurities

ANALYTICAL CHEMISTRY

Seeking Higher Resolution For Partials

11:30am Development & Validation of an In Vivo & In Vitro Potency Method for an AAV Gene Therapy

• Development of an In Vivo potency method with Western Blot quantification

• Surrogate functional activity determination

• Challenges with In Vivo expression of target, WB method validation and transfer

• Development of an In Vitro potency method to replace the In Vivo method

• Bridging of In Vivo results to the In Vitro method

Uditha deAlwis, Executive Director, Analytical Sciences, Sarepta Therapeutics

11:30am Rational Design & Development of Vector Genome Assay by ddPCR

• Design strategy for vector genome assay by ddPCR

• Comparability exercise and correlation with qPCR

• Pre/Qualification aspects and strategy

Santoshkumar Khatwani, Associate Director, Analytical Development, Sangamo

11:30am Capillary Electrophoresis & its Application to the Characterization of AAV Gene Therapy

• An overview of the application of CE based technics to the analysis AAV gene therapy product

• Case studies of CGE on capsid protein purity

• Case study on CGE on trans gene ID

• Case study on the development of CIEF based methods

Zack Zhang, Senior Scientist, Analytical Development, Sanofi

12:00 pm Session Reserved for ACF Bioservices

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12:00pm Session Reserved for Progen

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12:00pm Recent Advances in Precision Analytics for Oligonucleotide, Cell & Gene Therapy

 

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12:30pm Engineering Cell Lines for AAV Cell-Based Potency Assays

• Engineering cell lines, including reporter gene systems, can improve potency assay throughput, precsion and costs

• AAVR overexpressing cells can platform to nearly all serotypes and any transgene to improve permissivity and assay signal

• AAVR cells allow for stability-indicating potency assays with which to further our knowledge of AAV capsid structure-function relationships

• Our modular cell line engineering system can be applied to any new gene therapy product coming next in the pipeline

Stephanie Whipple, Scientist, Sanofi

12:30pm Validating Digital PCR & Safeguarding Comparability

• Comparison of qPCR v ddPCR for AAV titre analysis

• Other applications of ddPCR technology for AAV based gene therapies

• Overview of method validation requirements and discussion of site to site transfer

• Comparability assessment

• Overcoming throughput bottlenecks with the technology

Matthew Hankinson, Associate Director, Analytical Development, Allergan

12:30pm Analytical Ultracentrifugation for the Characterization of AAV-based Gene Therapy Products: From Method Development to a Release Assay

• Implemented an SV-AUC method for the full characterization of key quality attributes of AAV products all in one assay

• Developed a CsCl density gradient equilibrium method for the identification of unknown species in the c(s) distribution

• Applied standardization strategies to facilitate future transfer of the method to a QC lab

George Bou-Assaf, Senior Scientist, Protein & Gene Therapy Biophysical Characterization, Analytical Development – Product and Technology Development, Biogen

1:00pm Networking Lunch

2:00pm Exploring Data Software for Improved CellBased Assay & Parallelism Analysis

• Reviewing the different data analysis packages available for gene therapy analytical data

• Highlighting common pitfalls

• Analyzing parallelism data

Kendall Carey, Director, Analytical Sciences & Quality Control, Spark Therapeutics

2:00pm Roundtable Discussion: Molecular Biology Technologies

• Digital PCR

Sebastiaan van Zalen, Quality Control Manager, Clinical Vector Core (CVC), Children’s Hospital of Philadelphia

• Next Generation Sequencing

Jarrod Dean, Senior Scientist, Analytical Development, Sanofi

• Infectivity Assays

Ryan Burnett, Director, Analytical Development, Neurocrine

• Molecular Biology

Timothy Fenn, Director, Analytical Development, Prevail Therapeutics

2:00pm Method Development & Characterization of Partially Filled rAAV Vectors 

• Exploring analytical methodology for measuring partially filled capsids

• Discussing the characterization of partially filled capsids

• Considering the biological ramifications of rAAV products possessing higher populations of partially filled capsids

Michael Byrne, Senior Director, Analytical Development & Product Quality, REGENXBIO

2:30pm Session Reserved for Partner Organization 

2:30pm Session Reserved for Wyatt Technologies 

Print

3:00pm Potency Assay for Autologous Gene Editing Therapy

• Addressing mode of action

• Cell culture optimization

• Development of end points

Ricardo Borjas, Senior Scientific Fellow, Vertex

3:00pm Analytical Development Strategies for Detecting Product & Process Related Impurities

• Development landscape for residual assays

• Product vs process related impurities

• Regulatory and safety considerations (Sf9 vs HEK/ HeLa)

• Development of residual host cell DNA assays and residual host cell protein assay using a case study Bac Sf9 HCP development

Patrick Starremans, Associate Director, Analytical Sciences & Head of Process Analytics, Voyager Therapeutics

3:00pm Addressing Analytical Challenges in Gene Therapy Product Characterisation by LC-MS Method Development (REMOTE PRESENTATION)

• Regulatory requirements and challenges in gene therapy product characterisation

• Intact mass method development

• Peptide map method development: measuring CQA peptides

• Native MS for measuring full: empty ratios. Charge detection mass spectrometry

Francesco Lanucara, Senior Technical Specialist, Analytical Sciences, Allergan

3:30 pm Afternoon Refreshments & Networking

NAVIGATING GENE THERAPY MANUFACTURING CHALLENGES

4:00 pm Session Reserved for Bio-Rad

4:30 pm Panel Discussion: The Characterization & Consequences of Partially Filled Capsids

  • Yingmei Tao Director, Pharmaceutical Products Quality Control , Sarepta Therapeutics Yuan
  • Herbert Runnels Senior Research Fellow, Gene Therapy Global Analytical Sponsor , Pfizer
  • Michael Byrne Senior Director, Analytical Development & Product Quality , REGENXBIO
  • John Little Director, Quality Control, Freeline Therapeutics

Synopsis

• What different methods have been used to characterize partials?

• Where are some companies seeing success with partials?

• Do partials decoy the immune system or are they competitive with full capsids for target cells? How do partials affect potency?

5:00 pm Session Reserved for Covance

5:30 pm Characterization of Plasmid DNA Starting Materials

Synopsis

• Unraveling inverted terminal repeats

• Detection of cross-contamination

• Analysis of linearization efficiency

• Topology, the discussion that never ends

6:00 pm Chairs’ Closing Remarks

6:15 pm Poster Session & Happy Hour

Synopsis

The scientific poster session is an ideal opportunity to communicate your new results and expertise to a niche audience of industry experts, get feedback from peers and colleagues in the industry and learn how others in the field have been tackling similar challenges.