7:50 am Chair’s Opening Remarks

CMC CHALLENGES IN ANALYTICAL DEVELOPMENT

8:00 am Advances in Analytics to Streamline the Production of Viruses for Gene Therapy and Vaccination

Synopsis

  • Overview of analytical methods for characterisation of vaccines and gene therapy vectors’ critical quality attributes
  • Highlight of the main challenges in analytics to assist bioprocess development (UPS and DSP)
  •  Perspectives on integration of critical analytical tools in vaccine and gene therapy frameworks

8:30 am Speaking Position Reserved For Beckman Coulter

9:00 am Key CMC Challenges and Development Approaches for AAV Gene Therapy: Pre-Clinical to IND

Synopsis

  • The key CMC deliverables amid the pre-clinical to clinical phase of AAV gene therapy product development
  • Analytical challenges will be emphasised, including production yield vs. volume requirements, batch comparability, and stability

9:30 am Joint Q&A Session

10:00 am Morning Refreshments & Speed Networking

BIOASSAYS & MOLECULAR BIOLOGY

NAVIGATING THE CHALLENGES OF LIMITED MATERIALS

11:00 am Key Points in Developing qPCR and dPCR Based Methods for AAV Vector Quantification

Synopsis

  • Reference material characterisation
  •  Multiplexing in qPCR and Dpcr
  • A common step: sample preparation

11:30 am New Analytical and Biophysical Tools to Minimise Sample Consumption for Release and Characterisation Assays of AAV-Based Products

Synopsis

  •  AAV products comprise a protein capsid and a nucleic acid packaged inside.
  • The smaller manufacturing scales of AAV-based products make it particularly challenging to develop methods with very limited sample availability.
  •  We evaluate new technologies which require minimal sample amounts to inform critical quality attributes and we demonstrate how they are orthogonal to the traditional methods.

12:00 pm Joint Q&A Session

ANALYTICAL CHEMISTRY

DEFINING ANALYTICAL APPROACHES TO EXTENDED CHARACTERISATION

11:00 am PANEL DISCUSSION: Challenges of Extended Characterisation with Limited Samples

Synopsis

  •  Analytical approaches to extended characterisation
  • Applications of extended characterisation
  • Which Assays to use with limited sample availability
  • How Many are needed to provide sufficient data on key quality parameters for an acceptable production lot

11:30 am Extended Characterisation of Lentiviral Vectors: Closing the Gap with AAV

Synopsis

• Extended characterisation is required to demonstrate
product understanding of impurities and impact to efficacy.
• Lentiviral vectors (LVV) combine the complexity of AAV and
extracellular vesicles, and AAV techniques are not always
directly applicable to LVV.
• Case studies will be presented describing current methods
and future potential for LVV characterisation assays.

12:00 pm Joint Q&A Session

12:20 pm Lunch & Networking

CONTROL STRATEGIES & METHOD LIFE CYCLE MANAGEMENT

1:50 pm Future In-Vivo GT Control Strategy: Need for Adequate (Pre-) cQAs of AAV Based GT Products

Synopsis

  • Current and future GT control strategy
  • Assessing adequate CQA’s

2:20 pm The Positive Impact of Using AQbD Principles and Statistical Tools During Method Development

Synopsis

  • Benefits of using an ATP
  • First concept ideas how to characterise the MODR
  • Method control strategy

2:50 pm A QC Perspective: Method Life Cycle Management and Considerations for Gene Therapy Programs

3:20 pm Joint Q&A Session

CHARACTERIZATION OF AGGREGATES &
IMPURITIES

1:50 pm AAV Vectors: Product Related Impurities Characterisation

Synopsis

  •  Identifying product related impurities for AAV vectors
  • Analytical approaches to characterization of impurities

2:20 pm Control of Critical Analytical Reagents

Synopsis

Which analytical reagents should be considered critical analytical reagents?
This talk will discuss which and how they should be evaluated and managed
Dossier expectations at approval, and post approval changes

2:50 pm Extended Q&A Session

3:40 pm Afternoon Refreshments & Networking

CRITICAL QUALITY ATTRIBUTES IDENTIFICATION, ASSESSMENT & CONTROL STRATEGIES

4:10 pm HPLC and CE for AAV Critical Quality Attribute Characterisation

Synopsis

  • Gyroscope have developed QC-friendly methods for quantifying capsid titre, genomic titre, empty:full, aggregation using HPLC (SEC-MALS) and capsid protein purity (VP123) using CE for our clinical development programs.
  • As truly platform methods they give reliable, fast, low manual-handling results independent of AAV serotype that benefit both R&D and QC release strategies.

4:40 pm A Top-Down Approach to Gene Therapy Product Characterisation: Addressing Critical Quality Attributes from Intact Capsids to Amino Acid Modifications

  • Ian Anderson Associate Director Analytical Sciences, Allergan

Synopsis

  • Biophysical techniques for empty full and aggregation analysis
  • Intact mass and peptide map analysis for VP subunit characterisation
  • Multiple Attribute Monitoring: moving LC-MS from characterisation to QC release testing

5:10 pm Joint Q&A Session

5:30 pm Chairs’ Closing Remarks