8:50 am Chair’s Opening Remarks
BUILDING A PHASE APPROPRIATE ANALYTICAL STRATEGY
9:00 am Combinational Product Development – Bridging Science With Compliance
Synopsis
• Harmonised approaches to developing a successful global analytical strategy
• Quality based infrastructure development from concept to commercialisation
• Traversing the programmatic lifecycle (integrated approaches to the development of novel analytical methods and compedial applications)
9:30 am Measuring Strength of AAV Therapeutics
Synopsis
• Supporting drug discovery and drug development
• Quest for MoA-reflective methods
• Optimisation and automation of the infectious titre method
10:00 am Morning Refreshments
BIOASSAY & MOLECULAR BIOLOGY TRACK
POTENCY ASSAY STRATEGIC DEVELOPMENT
11:00 am (CASE STUDY) (NEW DATA) Quantity & Potency Assays for SVec, a Novel SV40 Based Viral Vector Platform
Synopsis
• Introduction to SVec as gene therapy vector and development of the VP2-qPCR assay to quantify vector genomes and the TCTD50-LUC potency assay to support initial development of the SVec production process
• Development of the Reverse transcriptase Q-PCR Potency Assay (RQPA), a relative potency assay for generic application.
• Product specific potency assays are presented for AMA005, Amarna’s lead product candidate targeting Hemophilia B
11:30 am (CASE STUDY) Potency Reference Standard Strategy: Laying a Clinical Foundation for Commercial Success
Synopsis
• Meeting regulatory expectations and overcoming the unique challenges presented by smaller scale clinical and commercial production
• Key aspects of planning and implementing a sound commercial two tiered reference standard strategy with a focus on robust potency methods
PHYSIOCHEMICAL TRACK
CHARACTERISING PRODUCT RELATED IMPURITIES
11:00 am Speaking Position Reserved for Bio-Techne
11:30 am Analytical Methods of AAV Vectors for Process Development & Quality Control
Synopsis
- Analytical methods for AAV for process development and for quality control will be introduced
- Pros and Cons of each method will be explained
- Current situation of analytical methods for AAV characterisations especially from scientific perspectives
12:00 pm Speaking Position Reserved for Refeyn
12:10 pm Lunch & Networking
ENHANCE YOUR GENOME SEQUENCING TO BETTER UNDERSTAND YOUR GENOME IDENTITY
1:00 pm (CASE STUDY) (NEW DATA) Quantitative Analysis of AAV Concentration and Integrity by Digital PCR
Synopsis
- Evaluation of multiple digital PCR platforms
- Optimisation and automation of PCR-based AAV titering
- Vector genome integrity by linkage analysis
1:30 pm (NEW TECH) (CASE STUDY) Genome Integrity Using DDPCR
Synopsis
- Definition of vector integrity: sequence variation or sequence fragmentation?
- Analytical assays for the characterisation
- Development of a 2D-ddPCR method for vector integrity determination
CHARACTERISING PRODUCT RELATED IMPURITIES
2:00 pm (CASE STUDY) Mass Spectrometric VP Characterisation of rAAV Gene Therapy Products
Synopsis
- Intro to our LCMS approach for Intact mass, peptide mapping, and PTM profiling
- Example of minor VP protein ID using combination of LC-MS and CE-SDS
- Example of PTM profiling to support formulation selection
2:30 pm Afternoon Refreshments & Networking
MASTERING AAV INFECTIVITY
3:00 pm (CASE STUDY) AAV Infectivity & Its Role in the Potency Matrix
Synopsis
• Development of a platform ddPCR-based method and comparability to TCID50
• Use cases for implementation and correlations to other potency readouts
3:30 pm (CASE STUDY) Relative Infectivity Assay Method
Synopsis
• Overview of current infectivity approaches
• REGENXBIO case study of relative infectivity approach, challenges and opportunities