*All Times Listed are in EST

8:00 am Chair’s Opening Remarks

DEFINING STANDARDS FOR A RAPIDLY EVOLVING FIELD

8:15 am The European experience on testing of Advanced Therapy Medicinal Products

Synopsis

• Discussing general expectations on analytical methods

• Highlighting the challenges/differences as compared to standard biological medicinal products

• Sharing experience gained in the context of the European early access program PRIME

8:45 am Committing to Harmonization to Reduce Variability & Improve Comparability: Setting Appropriate Standards

  • Yuan Zhao Principal Scientist, Leader of Gene Therapy Section, Advanced Therapy Division/ NIBSC, Medicines & Healthcare Products Regulatory Agency

Synopsis

• Challenges in standardization of gene therapy – historical efforts in standardization of gene therapy

• Challenges in developing standards for analytical assays

• Experience learnt from the development of the 1st WHO International Standard for gene therapy.

9:15 am Session Reserved for Beckman Coulter

9:45 am Panel Discussion: Building the Plane as They Fly It

  • Barbara Bonamassa Quality Assessor & European Medicines Agency (EMA) Expert for ATMPs, Italian Medicines Agency (AIFA)
  • Joseph Lee Executive Director, Analytical Development, Gene Therapy , PTC Therapeutics
  • Claire Davies Head of Bioanalytics , Sanofi
  • Stephen Duguay Head of Analytical Development, Bluebird Bio

Synopsis

• Early stage, late stage, and regulators perspectives on common analytical hurdles

• Where is there room novel technologies and surrogate assays?

• Q&A session

10:15 am Morning Refreshments & Networking

BIOASSAYS

Advances In Bioassays: Case Studies

MOLECULAR BIOLOGY

Implementing Next Generation Sequencing Technology

ANALYTICAL CHEMISTRY

Characterizing Aggregates

10:45am In Vitro Biopotency Assay of rAAV Vectors for Product Release

• Discussing the development of an in vitro biopotency assay for hemophilia

• Gene therapy AAV8

• Utilizing a HepG2 cell line

Johannes Lengler, Leader, Cell Culture & In Vitro Testing Group, Takeda

10:45am Comprehensive rAAV Genome Characterization Using Next Generation Sequencing (NGS) & its Application

• Comparison of current platforms (MiSeq/ PacBio/ Nanopore) for profiling rAAV genome

• Case study

• Validation of NGS workflow and analysis

Hui-wen Liu, Scientist I, Biogen

10:45am Next-Generation Aggregate Detection

• Navigating analytical challenges of working with AAV based gene therapeutics

• Platform methods for purity/impurity and aggregation determination of AAV based gene therapy product

• Highlighting relevant case studies

Julia Wang, Associate Director, AD-Analytical Chemistry, AveXis

11:15 am Session Reserved for Partner Organisation

11:15am Session Reserved for Partner Organisation

11:15am Protein or Not? Advanced High Throughput Aggregate Analysis with the Aura

• In protein-based formulations, distinguishing aggregated API from other particle types is important for understanding the root cause of instability and existing methods have been either unreliable or too cumbersome and difficult to use in many workflows

• Introducing the Aura, a 96-well low-volume aggregate and particle imaging system

• Exploring how the Aura can rapidly size, count, and characterize particles and identify them as proteins, non-proteins, hydrophobic, or other types of molecules

Rick Gordon, Vice President, Sales, Halo Labs

11:45am Advances in Immunoassays – What’s New?

• Highlighting the current challenges of working with immunoassays currently available for analytical scientists

• Exploring the advances in technology being implemented and anecdotal case studies

• Discussing vendors, new equipment and assays setups

Edward Rocnik, Director, Analytical Development, Takeda

11:45am Implementation Strategy of a NGS GMP Workflow

• Talk details to follow

Rachel Phair, Research Associate, Sanofi

11:45am Moving Complex Chemistry Methods into a QC Environment

• The success starts with a robust, QC friendly analytical method

• What method validation tells you

• Stability testing put the analytical methods to real test

Yingmei Tao, Director, Quality Control, Pharmaceutical Products, Sarepta

12:15pm Networking Lunch

1:15pm Development of a One-Step RT-ddPCR Method to Determine the Expression & Potency of AAV-based Gene Therapy

• One-step RT-ddPCR method has been developed to measure relative transcript levels

• Application of this method for in vitro determination of expression and potency of AAV vectors

• Application of this method for analyzing in vivo samples

• Correlation to other methods

Pete Clarner, Scientist I, Gene Therapy Accelerator Unit, Biogen

1:45pm Roundtable Discussion: Bioassays

Hannah Maheno, Scientist, Sanofi

Cecilia Della Valle, Associate Director, Analytical Science & Technology, Gene Therapy, CMC, PTC Therapeutics

Savita Sankar, Principal Scientist, Pfizer

1:15pm Forced Degradation Studies of AAV Biotherapeutics, Establishing the Applicability of Analytical Assays

• Forced degradation studies are a critical component of the CMC section in a BLA filing

• Here the AAV therapeutic was exposed to a series of degradation conditions (temperature, pH, oxidation and light) and studied using a range of analytical methods

• The purpose of this study is to determine the suitability of the analytical techniques employed during the drug development process

• Information from these studies can be used to correlate changes in structure with the function of the system

Matteo Placidi, Senior Scientist II, Analytical Sciences, Voyager Therapeutics

1:45pm Session Reserved for Ultragenyx

1:15pm Roundtable Discussion: Analytical Chemistry Techniques

Participate in a focused roundtable discussion to discuss key analytical chemistry technologies in an intimate environment. Each table will be led by an industry leader with experience in using the relevant methodology, so this is your opportunity to have your burning questions answered and benchmark your approach with your colleagues in the field. Roundtables will discuss the use of the common analytical chemistry technologies used for capsid characterization, including:

• High performance liquid chromatography (HPLC)

• Electrophoresis approaches, including capillary gel electrophoresis (CGE)

• Mass spectrometry (MS)

• Analytical ultracentrifugation (AUC)

Brian Hosken, Director, Analytical Chemistry, Sana Biotechnology

Sushmita ‘Mimi’ Roy, Executive Director, Analytical Sciences, Process Sciences, BioMarin

Amanda Werle, Principal Scientist, Analytical Research & Development, Pfizer

2:15 pm Afternoon Refreshments & Networking

Implementing High Throughput Technology To Adhere To Shorter Timelines

2:45 pm Automation: Powering the Next Generation of Therapeutics

Synopsis

Talk details to follow

3:15 pm A QC Perspective: Phase Appropriate Method Development & Validation for Gene Therapy Products

  • Lyndi Rice Head of QC Viral Vector Analytical , BioMarin

Synopsis

• CRO and CMO competition – a huge opportunity for third party service providers

• Saving time, reducing workloads, and improving technology transfer

• Leveraging communication to form beneficial relationships and encourage product ownership

3:45 pm Handling Pressure: Working to Shorter Timelines for Orphan Drugs & Optimizing Third Party Technology Transfer

  • Mark Galbraith Head of Quality Control & Analytical Sciences, Spark Therapeutics

Synopsis

• CRO and CMO competition – a huge opportunity for third party service providers

• Saving time, reducing workloads, and improving technology transfer

• Leveraging communication to form beneficial relationships and encourage product ownership

4:15 pm Chairs’ Closing Remarks